Dr. Frankel brings significant executive management experience in the development of successful therapeutics to Nocion as it continues the Phase 2b clinical trial of Taplucainium for chronic cough.
WATERTOWN, Mass.–(BUSINESS WIRE)–Nocion Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions”, that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain, today announced that it had appointed Matthew Frankel, M.D., MBA as Chief Medical Officer.
“We are delighted to welcome Matthew to the senior management team as we continue the clinical development of Taplucainium for chronic cough and the evolution of Nocion’s platform,” said Rick Batycky, CEO of Nocion Therapeutics. “Matthew brings important expertise and perspective from many years of large pharma medical management experience.”
“I am thrilled to join Nocion as we drive the ongoing Phase 2b refractory chronic cough trial forward to completion,” said Matthew Frankel, newly appointed CMO of Nocion Therapeutics. “I look forward to working with the team and the scientific community to help realize the vast potential of this platform which can silence nerves that cause the sensation of pain—while leaving touch and movement neurons unaffected.”
Matthew Frankel, M.D., MBA Before joining Nocion, Dr. Frankel served as Chief Medical Officer at Chemomab, Inc., where he spearheaded the development of a novel monoclonal antibody for fibrotic and inflammatory diseases, culminating in a successful end-of-phase 2 meeting with the FDA. Previously he was Vice President, Clinical Development & Medical Affairs, Specialty Pharma at Boehringer Ingelheim where he was responsible for developing new drugs for oncology, immunology, pulmonary, and CNS diseases. In a prior role at Novartis, Dr. Frankel was Vice President & Head, Immunology and Dermatology Medical Unit, where he oversaw medical affairs and clinical development for Cosentyx®, Ilaris®, and Zortress®. Earlier in his career, Dr. Frankel led the medical affairs unit at Sandoz supporting the biosimilar, biopharmaceutical and generics businesses. Dr. Frankel also previously held clinical development leadership roles across geographies and therapeutic areas at Reata, Fibrogen, Abbott Labs, and Schering Plough.
Dr. Frankel received a B.A. from Vassar College, an M.D. from the University of California School of Medicine, Los Angeles and an MBA from the J. L. Kellogg Graduate School of Management at Northwestern University. He is board certified in internal medicine.
ABOUT TAPLUCAINIUM Taplucainium (formerly NTX-1175) Dry Powder for Inhalation is a proprietary molecule in the novel class of CSCBs that allows for specific silencing of activated/inflamed nociceptors while having minimal local off-target effects or systemic exposure. Unlike other investigative cough therapies, such as P2X3 antagonists which target a specific Large Pore Channel (LPC), Taplucainium gains access to the nociceptor through any open LPC (P2X, TRPV, TRPA, etc.) whereupon it inhibits the sodium channels responsible for initiating the pathological cough response. This broad mechanism has shown significant antitussive effects in preclinical models of cough. Combined with good preliminary safety and efficacy data from earlier stage clinical work, this forms the basis for its use in cough indications beyond chronic cough.
ABOUT NOCION Nocion Therapeutics is a biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. Venture investors in Nocion include Arkin Bio Capital, Canaan Partners, F-Prime Capital, Lumira Ventures, Mass General Brigham Ventures, Mission BioCapital, Monograph Capital, Morningside, Osage University Partners, and an undisclosed strategic investor. For more information, visit: www.nociontx.com.
Data Expected in Q4 2025 from Study Evaluating the First Potential Therapy for Post-Viral Hyposmia
DELRAY BEACH, Fla., — Cyrano Therapeutics, Inc., a clinical-stage regenerative medicine company pioneering the development of treatments for smell loss, today announced that it has completed enrollment in the Phase 2 FLAVOR trial, a randomized, double-blinded, placebo-controlled, multi-dose clinical study evaluating CYR-064, its novel soft-mist nasal spray product candidate, for the treatment of post-viral hyposmia (smell loss). Data from the trial is expected in the fourth quarter of 2025.
The FLAVOR trial enrolled 150 patients across 14 clinical sites in the United States to assess the safety, tolerability, and efficacy of CYR-064 for the treatment for post-viral hyposmia compared to placebo over a six-month period. Post-viral hyposmia, a chronic sensory condition with no FDA-approved treatment, is an increasingly prevalent issue affecting more than 60 million people between the US, Europe and Japan, and millions more worldwide.
“Completion of enrollment in our FLAVOR trial is a significant milestone in our mission to develop the first potential pharmaceutical treatment for post-viral smell loss,” said Rick Geoffrion, President and CEO of Cyrano Therapeutics, Inc. “We are deeply grateful to the patients and investigators who have contributed to this critical research, and we look forward to analyzing the data, which will be instrumental in guiding the next phase of CYR-064’s development.”
CYR-064 is a patent-protected, intranasal formulation of a broad-spectrum phosphodiesterase (PDE) inhibitor designed to enhance olfactory neuron excitability and restore the sense of smell. A key component of CYR-064’s delivery system is Ursatec’s 3K® pumpsystem in combination with the Resyca soft mist spray technology, which enables precise, targeted deposition of the drug into the olfactory region. By utilizing a soft-mist formulation with controlled droplet size and low spray velocity, the 3K®-Ultra Soft system enhances drug deposition and absorption and increases the potential for therapeutic efficacy. This advanced delivery mechanism is designed to optimize patient outcomes while ensuring ease of administration.
“Completing enrollment in the FLAVOR trial is an important milestone in our efforts to bring forward the first potential treatment for patients suffering from post-viral hyposmia,” said Mas Takashima, MD FACS, Professor and Chairman of the Department of Otolaryngology – Head and Neck Surgery at Houston Methodist Hospital and the principal investigator for the FLAVOR trial. “We are encouraged by the commitment of the patients who participated and are eager to review the results and validate the promise of CYR-064 which has the potential to provide the first evidence-based treatment for patients suffering from post-viral hyposmia..”
About Cyrano Therapeutics Cyrano Therapeutics is a private, venture-backed clinical stage regenerative medicine company. Since our foundation, we have been working diligently to develop therapies for people struggling with the loss of smell and taste. To learn more, please visit cyranotherapeutics.com.
A first-of-its-kind collaboration in Canada between a philanthropic organization and a venture capital firm with a mission to invest in best-in-class cancer-focused companies.
Toronto, ON – April 10, 2025 – The Terry Fox Foundation (“Terry Fox”) and Lumira Ventures (“Lumira”) have partnered to launch The Cancer Breakthrough Fund (the “Fund”). There is a growing recognition of the need for cross-sector collaborations that can bridge the gap between research funded by governments and philanthropic organizations and private sector skills, expertise, and connectivity. This remains crucial to accelerate research towards commercialization through building and scaling transformative companies that can deliver impactful products for patients.
The Fund is that bridge and pairs the Terry Fox Foundation’s extensive network of leading cancer researchers pioneering scientific breakthroughs with Lumira’s deep investor networks and proven track record of building companies that deliver innovative therapies to patients. The Fund, which will be managed by Lumira Ventures, will invest in companies led by mission-driven entrepreneurs focused on the goal of delivering novel therapies to patients in the fight against cancer.
“In terms of real returns, the life sciences sector has outperformed all other sectors of the innovation economy for much of the past decade both in Canada and the US”, said Peter van der Velden, Managing General Partner, Lumira Ventures. ”Equally importantly, VC-backed healthcare companies have directly and indirectly accounted for over 50% of the novel products approved by the FDA and delivered to patients over the past decade. Unfortunately, despite that investment out-performance and patient impact, the sector has remained chronically under-funded in Canada. As a result, much of the value capture resulting from the building of life science companies based on Canadian innovation has largely accrued to foreign investors. As Canadians, we are committed to finding ways to support and celebrate innovators across our country. Our collaboration with the Terry Fox Foundation is, in our view, a best-in-class model for bringing more much needed catalytic capital to the process of building companies that can deliver transformative products for patients, create high-value jobs for Canadians, and provide significant returns for Canadian investors.”
The Fund will focus on investing in companies addressing some of cancer’s biggest challenges today. While survival rates have improved across certain cancer types, cancer remains the leading cause of death in Canada where it is estimated that 1 in 4 Canadians will die from cancer. Despite significant advances in innovative therapies and care, as global populations age, incidence of cancer worldwide is projected to grow by >75%, adding 35M new patients by 2050. As a result, investing in groundbreaking cancer treatments has never been more critical as it has the potential to save millions of lives and transform the future of healthcare.
“Over the last two decades, scientific discoveries and technological advances have helped us better understand the molecular drivers of cancer,” says Suman Rao, PhD, Principal, Lumira Ventures. “This has resulted in the development of a number of novel therapies improving survival rates across several cancers. However, given the complexity of the disease, there still remains a huge unmet need across multiple cancer types.”
“In the forty-five years since Terry’s Marathon of Hope, the Terry Fox Foundation has been making innovative, transformational moves in our approach to funding cancer research – a reflection of the bold mission Terry set out to achieve,” says Michael Mazza, Executive Director, Terry Fox Foundation. “This partnership with Lumira Ventures is an evolution of the traditional philanthropic model in medical research, bringing together the best of charitable organizations and the private sector to accelerate progress and help finish Terry’s fight against cancer.”
For more information about the Fund and how to get involved either as a donor or as an investor, please contact Michael Mazza (CEO, Terry Fox Foundation, michael.mazza@terryfox.org) or Peter van der Velden (Managing General Partner, Lumira Ventures, plv@lumira.vc).
– 30 –
About Lumira Ventures
Lumira Ventures is a leading life sciences venture capital firm which has a long and deep track record of partnering with like-minded investors and mission driven entrepreneurs to build and scale transformative companies that positively impact patient lives, while generating strong returns for its investors. In the past 6 quarters, Lumira has participated in $1.4B of financings, added 8 new companies to its portfolio, enjoyed 5 exits/realizations and its portfolio companies have received 5 FDA approvals for novel therapies – three of them targeting cancer. Lumira is actively raising the strategic Cancer Breakthrough Fund in parallel with its core fund, Lumira Ventures Fund V.
About The Terry Fox Foundation Terry Fox Foundation honours the vision and spirit of an iconic Canadian while raising critical funds for cancer research. As a leading national charitable organization, the Terry Fox Foundation plays a vital role in building community, engaging more than 20,000 passionate volunteers and 3.5 million students in nearly 10,000 annual fundraising events across the country. People around the world of all ages, backgrounds, and abilities have been inspired by Terry’s enduring legacy. Through the generous support of our donors, partners, and volunteers, the Terry Fox Foundation has raised more than $950 million and funded over 1,300 innovative cancer research projects, bringing hope and health to millions of Canadians. Visit terryfox.org for more information and to learn how you can help take Terry’s Marathon of Hope across the finish line.
Puzzle Medical Devices is Thrilled to Announce the Closing of its CAN$43 Million Round to Complete its Clinical Study Led by KF Matheson and Desjardins Capital
Montreal, QC – Puzzle Medical, a Montreal-based medtech startup developing a percutaneous heart pump to support cardiac and renal function in patients with advanced heart failure, today announced the successful closing of a CAN$43 million funding round. The round was led by KF Matheson and Desjardins Capital, with participation from Lumira Ventures, Longview Ventures, BDC Capital, and existing investors.
This raise represents one of the largest medical device raises in the past two years in Canada. The capital will be used to refine Puzzle Medical’s percutaneous heart pump and its novel features, advance clinical studies to assess its safety and efficacy, and scale the team to support the company’s growth. As part of this milestone, Puzzle Medical is pleased to welcome new board members Kris Shah and David Prim, as well as board observers Daniel Hetu and Steven Abrams.
“Given the current macroeconomic landscape, this funding is a true testament to the impact of our work, the dedication of our team, and the unwavering support of our investors,” said Jade Doucet-Martineau, CEO and co-founder of Puzzle Medical. “These funds will accelerate our mission to transform the treatment of advanced heart failure.”
”The innovative technology being developed by Puzzle Medical will have a positive impact on cardiac patients worldwide. The Puzzle team and their achievements to date are an exemplification to the talent and ingenuity of Canadian entrepreneurs. We are proud to support their important work” – Frank Baylis, co-founder of KF Matheson.
“At Desjardins, we firmly believe in innovation and the good it can do for patients’ health. Desjardins Capital’s investment in Puzzle Medical Devices via the CRCD is in step with our commitment to supporting the growth of companies that create revolutionary technology. As a proud partner since February 2023, we’re thrilled to work with Puzzle Medical Devices to improve treatment of heart failure and offer patients worldwide better quality of life”. – Nathalie Bernard, Chief Operating Officer, Desjardins Capital.
“Our investment in Puzzle continues our long history of partnering with entrepreneurs dedicated to building world-class cardiovascular medical device companies. We are excited to support Puzzle’s team in the development of their breakthrough technology which has the potential to significantly impact the treatment of patients with heart failure.” – Daniel Hétu, Managing Director of Lumira Ventures.
About Puzzle Medical Devices
Founded in 2018, Puzzle Medical Devices is dedicated to improving patient outcomes through the development of a percutaneous heart pump for patients with advanced heart failure. The device’s modular design allows for safe implantation to support both renal and cardiac function through 4mm-pumps anchored in parallel in the descending aorta. For more information, visit puzzlemed.com.
Disclosure: Puzzle Medical’s device is not approved by the FDA and is not for sale in any countries.
About KF Matheson
KF Matheson serves as the family office of Frank Baylis and Kris Shah, two long-standing Canadian business partners and accomplished medical device entrepreneurs. Together, they have co-led and successfully exited multiple ventures in the medical device sector, with a combined transaction value exceeding CAN$2 billion. Leveraging its accumulated capital, the firm enables permanent, sustained impacts aimed at advancing quality of life worldwide. Its medical device portfolio seeks to improve the lives of patients through the conception and commercialization of state-of-the-art medical devices.
About Desjardins Capital
Backed by 50 years’ expertise, Desjardins Capital’s mission is to contribute to the prosperity of individuals and communities by investing in the growth of Quebec businesses. With assets under management of $3,2 billion as of December 31, 2024, Desjardins Capital contributes to the longevity of nearly 730 companies, cooperatives and funds in various sectors of activity from all regions of the province. In addition to contributing to socio-economic development, productivity and the adoption of sustainable practices, this subsidiary of Desjardins Group offers entrepreneurs access to a broad business network, enabling them to be accompanied and supported in their projects.
About Lumira Ventures
Lumira Ventures is the largest and most active life sciences venture capital investor domiciled in Canada. Lumira invests in best and/or first-in-class innovative healthcare companies that are primarily located in “secondary” geographies within North America. Its goal is to partner with mission-driven entrepreneurs to develop transformative therapeutics products and medical devices while generating strong financial returns for our investors and meaningful impact for patients. To that end we are proud to share that our portfolio companies have received FDA approvals for over 40 products that are improving the lives of over 1 billion patients worldwide.
About Longview Ventures
Longview Ventures is an independent investment vehicle that makes investments in select portfolio companies of Broadview Ventures, a mission-driven investment organization focused on cardiovascular disease and stroke. Longview focuses on clinical-stage companies as a complement to Broadview’s continued dedication to Seed and Series A financings. Longview and Broadview are funded by the Leducq Charitable Trust.
To fund completion of the EMPOWER Trial U.S. pivotal study and continue the commercialization of the Carillon Mitral Contour System
KIRKLAND, Wash., March 20, 2025 — Cardiac Dimensions®, a leader in minimally invasive treatments for heart failure and functional mitral regurgitation (FMR), today announced the close of an oversubscribed $53 million Series E financing round led by Ally Bridge Group with significant participation from existing investors. This financing will fund the completion of the EMPOWER Trial U.S. pivotal study and support the continued commercialization of the innovative Carillon Mitral Contour System®.
“Cardiac Dimensions is uniquely positioned to provide a safe, simple, and effective solution for heart failure patients suffering from FMR. A financing of this magnitude reflects the strong conviction of Ally Bridge, as well as our existing insiders, in the Carillon® therapy’s ability to play a central role in the treatment of this high-need patient population,” said Rick Wypych, president and CEO of Cardiac Dimensions.
“With these additional resources, we are also well-positioned to continue our global commercial expansion of the Carillon therapy to improve the lives of patients with this debilitating disease,” continued Wypych.
“We are excited to lead this financing to help support Cardiac Dimensions’ significant advancements in the treatment of heart failure patients with FMR,” said Steve Plachtyna of Ally Bridge Group and new member of the company’s board of directors. “We have been very impressed with the progress the company has made over the past several years in both their clinical efforts and their expansion of commercial sales outside the U.S. This financing will fund and accelerate both of these efforts.”
The Carillon device is designed to restore natural mitral valve function without damaging the mitral valve leaflets. The simple, catheter-based procedure works by restoring the valve’s natural function and promoting favorable left ventricular remodeling. Clinical studies have shown the Carillon therapy reduces mitral regurgitation, improves quality of life, and has extended survival for a broad range of patients with FMR. Furthermore, patients with early-stage FMR may be treated with Carillon therapy as a front-line treatment option, as it does not prevent the use of other future therapies.
Functional mitral regurgitation is a prevalent condition among individuals with heart failure, which affects over 64 million people globally.1 Studies indicate that moderate or greater severity of FMR is present in up to 59% of heart failure patients.2 When left untreated, FMR can lead to increased hospitalization due to heart failure, reduced quality of life and higher mortality rates.
About Cardiac Dimensions Cardiac Dimensions® is a leader in the development of innovative, minimally invasive treatment modalities to address heart failure and associated cardiovascular conditions. The company’s flagship technology, the Carillon Mitral Contour System®, is designed to address functional mitral regurgitation (FMR) using a catheter-based approach. Cardiac Dimensions has operations in Kirkland, Washington, Sydney, Australia and Frankfurt, Germany. For more information, visit www.cardiacdimensions.com.
Cardiac Dimensions, Carillon, and Carillon Mitral Contour System are registered trademarks of Cardiac Dimensions.
The National Venture Capital Association (NVCA) has named Lumira Ventures portfolio company HistoSonics as the recipient of its prestigious 2025 Startup Innovator Award.
“HistoSonics is a shining example of the entrepreneurial ecosystem at its best,” said NVCA President and CEO Bobby Franklin. “It all started with groundbreaking research at the University of Michigan, followed by dedicated entrepreneurs and investors working together to bring that innovation to market. In collaboration with government medical and regulatory agencies, this incredible technology is now transforming tumor treatment—offering patients a way to fight the disease without the harsh side effects of traditional methods. By changing the paradigm of health care with this cutting edge technology, HistoSonics is making a profound impact on both science and society, and we are proud to name them our 2025 NVCA Startup Champion.”
NVCA will celebrate HistoSonics at the 2025 NVCA Leadership Awards Dinner. The event will take place on June 5, 2025, at the Rosewood Hotel in Menlo Park, CA.
This award recognizes a VC-or growth equity-backed portfolio company that has made a positive and measurable impact on addressing an area of need, an ongoing crisis, or a significant challenge that exists in our world.
About Histosonics
HistoSonics is a privately held medical device company developing non-invasive platforms and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. For more information about Histosonics, please visit www.histosonics.com
About The National Venture Capital Association (NVCA)
The National Venture Capital Association (NVCA) empowers the next generation of American companies that will fuel the economy of tomorrow. As the voice of the U.S. venture capital and startup community, NVCA advocates for public policy that supports the American entrepreneurial ecosystem. Serving the venture community as the preeminent trade association, NVCA arms the venture community for success, serving as the leading resource for venture capital data, practical education, peer-led initiatives, and networking. For more information about NVCA, please visit www.nvca.org.
Agreement Provides Cyrano Therapeutics with Upfront and Milestone Payments, Plus Royalties from Commercialization of CYR-064 in Japan
DELRAY BEACH, Fla., Feb. 18, 2025 /PRNewswire/ — Cyrano Therapeutics, Inc., a clinical-stage regenerative medicine company pioneering the development of treatments for smell loss, announced today that on February 14, 2025 it entered into an option agreement with KYORIN Pharmaceutical Co., Ltd. for CYR-064, a novel treatment for post-viral loss of smell (hyposmia).
Under this agreement, KYORIN obtains an option for the license of the right to develop and commercialize CYR-064 in Japan and will pay an option fee to Cyrano. Upon exercising the option and entering into a licensing agreement, KYORIN will provide Cyrano with an upfront payment, milestone payments tied to the progress of developing and commercializing CYR-064, and royalties based on a specified percentage of net sales following its launch.
CYR-064, a novel, patent-protected, intranasal formulation of a broad-spectrum phosphodiesterase (PDE) inhibitor theophylline, is currently being administered in a Phase 2 clinical trial in the United States, with data from the trial expected in the second half of 2025. CYR-064 targets the inhibition of cAMP degradation thereby increasing intracellular concentrations and increasing olfactory neuron excitability, leading to a potential therapeutic effect in the treatment of post-viral hyposmia and potentially other olfactory disorders.
Post-viral hyposmia is defined as a persistent olfactory dysfunction after an upper respiratory tract infection, even after the upper respiratory inflammation has subsided. A virus is the most common cause of olfactory dysfunction, followed by chronic rhinosinusitis, Parkinson’s disease and head trauma. With the loss of smell, patients also lose up to 80% of their perception of taste. Forty percent of patients with olfactory dysfunction also experience more than two safety events and 43% experience depression3, significantly reducing a patient’s quality of life. At present, there are no medications with clear evidence of effectiveness in treating post-viral hyposmia.
About Hyposmia Hyposmia, including post-viral hyposmia, is an increasingly prevalent and serious chronic sensory condition for which there is no approved drug therapy and limited treatment options. Hyposmia causes significant impairment in quality of life for many sufferers. Moreover, in older individuals, hyposmia is associated with an increased risk of cognitive impairment and mortality. There is currently no FDA-approved therapy indicated for the treatment of smell loss.
About KYORIN Pharmaceutical Co. Ltd. KYORIN Pharmaceutical Co., Ltd. was founded in 1923. Accelerating its evaluation and acquisition of in-licensed products and establishing a presence in designated fields, KYORIN aims to contribute broadly to people’s health by pursuing innovation in drug discovery, in order to strengthen drug discovery capability and create high-value new drugs that meet medical needs. https://www.kyorin-pharm.co.jp/en/
About Cyrano Therapeutics Cyrano Therapeutics is a private, venture-backed clinical stage regenerative medicine company. Since its foundation, Cyrano has been working diligently to develop therapies for people struggling with the loss of smell and taste. To learn more, please visit cyranotherapeutics.com
Clinical practice guidelines for olfactory dysfunction (The Japanese rhinologic society) Japanese Journal of Rhinology2017, 56, 487-556, 2025, 64, 1-85
Coelho et al. Am J of Otol.: 2021 Jul-Aug;42(4):103001
Damona Pharmaceuticals, a clinical-stage biopharmaceutical company focused on discovering and developing small molecules for the treatment and prevention of cognitive deficits associated with brain disorders, announced today the publication of data demonstrating that DPX-101 reverses cognitive deficits induced by amyloid deposition in a widely used preclinical model of Alzheimer’s disease. The publication, “Procognitive and Neurotrophic Benefits of α5-GABA-A Receptor Positive Allosteric Modulation in a β-Amyloid Deposition Mouse Model of Alzheimer’s Disease Pathology,” will be published in the March 2025 issue of Neurobiology of Aging and can be viewed online.
“Current approved drugs for Alzheimer’s disease slow decline, but none of them reverse lost cognitive functions,” said John Reilly, CEO of Damona. “These preclinical results highlight the potential of DPX-101 to improve cognitive deficits while also providing disease-modifying effects in Alzheimer’s disease. The data provide an important proof of concept for our novel approach to restore cognitive functions associated with brain disorders by precisely targeting the α5 subunit of the GABA-A receptor to augment its function and restore contacts between neurons. We look forward to advancing our best-in-class DPX-101 program into clinical development toward our goal of restoring optimal brain function for patients suffering from early-stage Alzheimer’s disease as well as the treatment of cognitive impairment in depression and schizophrenia.”
Reduced levels of somatostatin (SST) and reduced numbers of SST-expressing GABAergic neurons (critical inhibitory neurons in the central nervous system) have been linked to cognitive deficits—impairments in the ability to understand, remember, and think clearly—in Alzheimer’s disease and other brain disorders. SST cells inhibit pyramidal cell dendrites mainly through α5-GABA-A receptors (α5-GABAA-R). Positive allosteric modulation of these receptors has shown cognitive and neurotrophic benefits in disease models of stress and aging. DPX-101 is a receptor-positive allosteric modulator (PAM) designed to target the α5 subunit of the GABA-A receptor selectively to augment its function and restore contacts between neurons. This study evaluated the ability of DPX-101 to prevent cognitive deficits and neuronal spine loss in the early stages of β-amyloid deposition and to reverse them in late stages in a widely used preclinical model of Alzheimer’s disease.
Summary and key findings: Damona’s preclinical small molecule DPX-101 improved working memory deficits and prevented neuronal shrinkage and spine loss in early and late stages of β-amyloid deposition in the 5xFAD mouse model of Alzheimer’s disease-related β-amyloid pathology. Specifically:
Acute administration of DPX-101 prevented spatial working memory deficits at 2 months of age
Chronic administration of DPX-101 reversed spatial working memory deficits at 5 months of age
Chronic administration of DPX-101 prevented pyramidal cell atrophy
Chronic administration of DPX-101 preserved pyramidal cell spine density, spine count, and dendritic length at early and late time points, despite ongoing β-amyloid accumulation
The cognitive and neurotropic effects of DPX-101 occurred independent of β-amyloid buildup
About DPX-101 DPX-101 is an investigational positive allosteric modulator (PAM) that selectively targets the α5 subunit of the GABA-A receptor (α5-GABAA-R) to enhance its function. The α5-GABAA-R subtype is a promising target for reversing cognitive impairment due to its location in the hippocampus and frontal cortex regions of the brain and its unique role in somatostatin-containing GABAergic interneuron tonic inhibition within microcircuits that are critical for healthy cognition. In preclinical studies, DPX-101 demonstrated highly selective activation of the α5-GABAA-R and a broad therapeutic window with no side effects. In preclinical models of stress, depression, aging, and Alzheimer’s disease, DPX-101 reversed memory deficits and restored connections between brain cells. In a preclinical model of schizophrenia, DPX-101 reversed hyperactivity of dopamine neurons, a hallmark of schizophrenia. DPX-101 is orally available, passes the blood-brain barrier, and achieves appropriate plasma and brain levels.
About Damona Pharmaceuticals Damona Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to discovering and developing transformational precision medicines for patients suffering from cognitive deficits associated with brain disorders. Damona’s approach combines deep expertise in brain microcircuits with precision chemistry and targeted neuropharmacology to develop small-molecule therapeutics that restore optimal brain function. The company’s scientific founder—Etienne Sibille, PhD, Scientific Director of the Neurobiology of Depression and Aging Program at the Centre for Addiction and Mental Health (CAMH) in Toronto—and collaborators have made pioneering discoveries regarding the unique role of the α5-GABA-A receptor subtype in cognition and demonstrated its potential for reversing cognitive deficits in models of multiple brain disorders. Damona’s lead clinical program, DPX-101, is designed to reverse cognitive impairment and restore connections between brain cells. Learn more at www.damonapharma.com.
Contact: Mary Moynihan M2Friend Biocommunications +1 (802) 951-9600 mary@m2friend.com
Cyrano Therapeutics, Inc., a clinical-stage regenerative medicine company pioneering the development of treatments for smell loss, announced today that it is providing clinical material to support an investigator-led feasibility study evaluating CYR-064 in Parkinson’s Disease patients.
CYR-064, Cyrano Therapeutics’ lead product, is also currently being evaluated for the treatment of patients suffering from hyposmia and anosmia with various causes, including post-viral hyposmia, in a 150-subject, randomized placebo-controlled Phase 2 clinical study being conducted at 14 sites in the U.S. Top-line data from this trial is expected in the second half of 2025.
Hyposmia and anosmia, the partial or complete loss of smell, affect approximately 95% of the one million individuals in the U.S. living with Parkinson’s disease, often serving as one of the earliest symptoms of the condition. Loss of smell typically precedes motor symptoms by five or more years, significantly impacting quality of life by diminishing the perception of taste, contributing to reduced appetite, and leading to weight loss—a common challenge for Parkinson’s patients.
“The potential to restore the sense of smell in Parkinson’s patients represents a significant step forward in addressing an often-overlooked aspect of the disease that decreases a patient’s quality of life and contributes to the decline of their nutritional health,” said Rick Geoffrion, co-founder and CEO of Cyrano Therapeutics. “We are excited to support this important investigator-initiated study, which aligns with our mission to improve the lives of individuals living with chronic loss of smell and the associated loss of taste perception.”
The new study, titled “A Single Arm Feasibility Study for the Treatment of Parkinson’s Disease Related Hyposmia Using CYR-064 Theophylline Spray” (Protocol No. CYR-064-12), is being conducted under the leadership of David Silvers, MD, FAAN, in Palm Beach Gardens, Florida. The 32-week study will enroll approximately 15-20 adult patients aged 18 to 80 years with moderate to severe hyposmia due to Parkinson’s disease. The study protocol includes a 24-week treatment period during which patients will receive a twice daily dose of CYR-064 through two sprays per nostril.
The purpose of the study is to evaluate the feasibility, safety, and potential effectiveness of CYR-064 in improving the sense of smell and taste perception in affected individuals. CYR-064 delivers theophylline, which is commonly used in the treatment of asthma and chronic obstructive pulmonary disease (COPD). While theophylline is FDA-approved for oral and intravenous use in COPD, systemic delivery of the drug causes side effects and drug interactions which limit its potential as a therapy for smell loss. CYR-064 is a novel formulation intended for intranasal administration with a proprietary spray device technology enabling targeted delivery to the olfactory region.
“Hyposmia is a challenging and debilitating symptom for many Parkinson’s patients, and innovative approaches like CYR-064 are critically needed,” said Dr. Silvers, principal investigator of the study. “We look forward to assessing this promising therapy’s potential to address a significant unmet need in Parkinson’s care.”
This investigator-initiated study represents Cyrano Therapeutics’ commitment to expanding the therapeutic potential of CYR-064 beyond post-viral smell loss, building on the company’s robust clinical program.
About Hyposmia Hyposmia, including post-viral hyposmia, is an increasingly prevalent and serious chronic sensory condition for which there is no approved drug therapy and limited treatment options. Hyposmia causes significant impairment in quality of life for many sufferers. Moreover, in older individuals, hyposmia is associated with an increased risk of cognitive impairment and mortality. There is currently no FDA-approved therapy indicated for the treatment of smell loss.
About Gardens Neurology, Palm Beach Gardens, FL Investigator: Dr. David Silvers, Board Certified in Neurology, Electrodiagnostic Medicine/EMG and is a Diplomate in Neuromuscular Medicine as well as a Diplomate in Behavioral Neurology and Neuropsychiatry. Gardens Neurology is accepting new patients and second opinion consultations. To make an appointment please call (561) 799-2831
About Cyrano Therapeutics Cyrano Therapeutics is a private, venture-backed clinical stage regenerative medicine company. Since our foundation, we have been working diligently to develop therapies for people struggling with the loss of smell and taste. To learn more, please visit cyranotherapeutics.com
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CNP-103, a nanoparticle in development to address the underlying autoimmunity of Type 1 diabetes (T1D).
“Clearance of the IND application for CNP-103 is a notable moment for COUR, as CNP-103 will be our third proprietary autoimmune disease program developed using our nanoparticle platform for antigen-specific immune tolerance to enter the clinic,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-103 has demonstrated the ability to stop T1D disease progression while creating an enhanced pro-regulatory environment with reduced inflammatory cell activity. These findings lead us to believe that CNP-103 could potentially preserve β-cell function and reverse dysglycemia, which are results that have also been observed in preclinical models. We expect to initiate a Phase 1b/2a first-in-human trial of CNP-103 later this year.”
The Phase 1b/2a study will assess the safety of CNP-103 in adults (aged 18-35) and pediatrics (aged 12-17) who have Stage III or newly diagnosed (within the last 6 months) T1D as well as C-peptide >0.2 ng/mL.
About CNP-103:
CNP-103 is a biodegradable nanoparticle encapsulating four recombinant proteins known to promote islet cell destruction: preproinsulin, GAD65, IGRP, ZnT8. Notably, these proteins cover >95% of known antigens driving Type 1 diabetes. By inducing tolerance to these proteins, COUR aims to prevent islet cell destruction by pathogenic CD4+ and CD8+ T cells, allowing for maintenance of insulin production and potential reversal of dysglycemia.
About T1D:
T1D is a progressive disorder impacting >1.5 million individuals in the U.S. and is caused by autoreactive pathogenic CD4+ and CD8+ T cells targeting insulin-producing β-cells in the islets of Langerhans, causing destruction. Progressive β-cell loss results in insulin deficiency and inability to regulate glucose. People who live with T1D experience symptoms that include acutely frequent urination, excessive thirst, weight loss, fatigue, and potentially life-threatening diabetic ketoacidosis. Broader health impacts of T1D include heart disease, kidney failure, stroke, and vision loss.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is enrolling patients in a Phase 1b/2a clinical study in Myasthenia Gravis (MG) and expects to initiate two trials in 2025: a Phase 1b/2a in Type 1 Diabetes and a Phase 2b in Primary Biliary Cholangitis (PBC), a disease in which COUR has already shown positive results in a Phase 1b/2a study. Additionally, COUR has partnered with Takeda Pharmaceuticals for its program in Celiac Disease, which is currently enrolling in a Phase 2b trial and is developing an undisclosed preclinical stage program in collaboration with Genentech.
Sound Blade Medical, Inc., a medical device company developing handheld ultrasound-guided histotripsy technology, announced today the successful closing of an oversubscribed US $16.5 million Series A funding round. Amzak Health and Lumira Ventures co-led the round, with participation from Invest Nova Scotia. Funds will be used to accelerate the development and clinical validation of its pioneering technology designed to bring the benefits of precision ultrasound therapy to a wide range of patient conditions.
“We’re thrilled to have the support of such forward-thinking investors who share our vision for the future of Sound Blade and our technology,” said Jeremy Brown, Ph.D., Co-founder and CEO of Sound Blade. “This funding enables us to rapidly advance our technology, expand our team and accelerate regulatory approvals to quickly bring our therapy to patients in need.”
Histotripsy is a non-invasive, non-ionizing and non-thermal ablation technology guided by real-time imaging that uses focused ultrasound delivered from outside the body to mechanically destroy targeted tissue. It works by inducing cavitation bubbles in the tissue at the ultrasound beam focus, using short, high-pressure pulses. When the bubbles collapse, only liquified tissue remains. Because of the non-thermal nature of the technology, it may allow better immune and healing responses and avoid dangers associated with heating. In comparison to open surgery, histotripsy offers the possibility of dramatically improved patient outcomes including less blood loss, fewer complications, reduced recovery time, and decreased chance of infection.
Tony Natale, MD, Partner at Amzak Health, stated, “Sound Blade’s handheld histotripsy has truly disruptive potential. We’re combining a highly accomplished founding team with an investor syndicate that has built many successful Medtech companies to unlock the full promise of this platform.”
Gerry Brunk, Managing Director at Lumira Ventures, commented, “We’re excited to continue our long history of backing world-class medical device companies in Canada. With its roots at Dalhousie University and Nova Scotia Health, Sound Blade represents Lumira’s second investment in the exciting field of histotripsy for the non-invasive treatment of important diseases.”
About Sound Blade Medical Founded in 2023, Sound Blade Medical is at the forefront of developing advanced handheld histotripsy technology with world-leading precision. Its mission is to improve patient outcomes through groundbreaking non-invasive solutions. For more information, visit https://soundblademedical.com/.
Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) is pleased to provide a business update regarding the successful completion of the regulatory review process for GRAFAPEX™ (treosulfan) for injection with the US Food and Drug Administration and, in addition, to announce preliminary estimates of the company’s operating and financial results for the company’s third fiscal quarter ended December 31, 2024 (which remain subject to completion of Medexus’s financial closing procedures). All dollar amounts in this news release are in US dollars unless specified otherwise.
FDA approval of GRAFAPEX™ (treosulfan) for injection
On January 22, 2025, Medexus was informed that the FDA approved GRAFAPEX™, an alkylating agent, with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients one year of age and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). GRAFAPEX™ holds Orphan Drug Designation under the Orphan Drug Act, meaning that the product will benefit from up to seven-and-a-half years of regulatory exclusivity in the FDA-approved indication. Medexus holds exclusive commercial rights to GRAFAPEX™ in the United States under a February 2021 exclusive license agreement with medac GmbH.
“We are pleased to report this positive development, which marks a strategically important step forward for our business and, importantly, will now benefit eligible patients across the United States,” commented Ken d’Entremont, Medexus’s Chief Executive Officer. “Not only will GRAFAPEX™ make a substantial contribution to alloHSCT in the United States, but it also solidifies Medexus’s leadership position in this therapeutic field.”
“We are targeting a commercial launch in the first half of calendar year 2025, and given our recent experience in Canada we are very optimistic about the potential of GRAFAPEX™ in the US market,” added Richard Labelle, Medexus’s Chief Operating Officer. “We anticipate that GRAFAPEX™ will have a meaningful impact on Medexus’s total revenue and believe that annual product-level revenue in the United States has the potential to exceed US$100 million within five years after commercial launch.”
“This FDA approval provides a useful option for adult and pediatric patients, with the potential to enhance overall survival while minimizing side effects,” said Dr Filippo Milano, a stem cell transplant physician-scientist and principal investigator in clinical trials using treosulfan as part of a conditioning regimen.
As previously announced, the regulatory milestone amount payable to medac under the fourth amendment to the February 2021 exclusive license agreement between the parties is based on the language of the product label approved by the FDA. Based on the terms of the approval, including the FDA-approved product label, Medexus has determined that medac will earn a $15 million regulatory milestone amount. The final amount is subject to review and confirmation by the parties in light of the terms of the agreement. The regulatory milestone amount will be payable in installments, subject to Medexus’s right to temporarily defer installment amounts, on terms described in Medexus’s December 2, 2024 press release, available via the Investors section of Medexus’s corporate website. For a $15 million regulatory milestone amount, this installment schedule will result in payments of US$2.5 million by June 30, 2025, US$5 million by October 1, 2025, and US$7.5 million by January 1, 2026 – subject to Medexus’s temporary deferral option in respect of the second and/or third such payments. Also as previously announced, given the FDA approval of GRAFAPEX™, Medexus will now promptly repay a US$2.5 million credit received from medac in September 2021.
Additional information about the terms of the license agreement, including copies of the relevant documents, is included in the company’s filings on SEDAR+ at www.sedarplus.ca. The summary in this news release is qualified by reference to the terms of each such document as applicable.
Preliminary estimates for fiscal Q3 2025
Medexus remains focused on delivering strong revenue growth and overall performance across the company’s entire portfolio of products in both the United States and Canada. This important new development for Medexus regarding GRAFAPEX™ arrives on the heels of what is expected to be a solid fiscal Q3 2025, and includes an estimated $1.9 million in fiscal Q3 2025 investments in personnel and infrastructure that were made to prepare for this recent positive FDA decision.
Medexus currently expects key selected highlights for fiscal Q3 2025 to include the following –
Revenue between $29.5 million and $30.5 million for the three-month period ended December 31, 2024, representing a year-over-year increase of at least 17% over $25.2 million for fiscal Q3 2024, attributable in part to continuing growth in net sales of Rupall, and an approximately $2.0 million beneficial impact of customer buying patterns and related timing of orders of IXINITY relative to patient unit demand in fiscal Q3 2025.
Adjusted EBITDA between $5.5 million and $6.0 million for the three-month period ended December 31, 2024, representing a year-over-year increase of at least 70% over $3.2 million for fiscal Q3 2024, primarily attributable to the effects of the company’s ongoing financial discipline efforts, together with the effect of customer buying patterns mentioned above, and partially offset by the estimated $1.9 million of GRAFAPEX™ personnel and infrastructure investments mentioned above. (Refer to “Non-GAAP measures” at the end of this press release for information about Adjusted EBITDA.)
Available liquidity of approximately $8.5 million (December 31, 2024), consisting of cash and cash equivalents, compared to $5.3 million (March 31, 2024).
Operating income between $3.5 million and $4.0 million for the three-month period ended December 31, 2024, representing a year-over-year increase of at least 123% over $1.6 million for fiscal Q3 2024.
Net income between $(0.5) million and $2.0 million for the three-month period ended December 31, 2024, compared to $(0.5) million for fiscal Q3 2024. Final reported net income for fiscal Q3 2025 will depend on completion of Medexus’s financial closing procedures, including in respect of current and deferred income tax expense amounts.
The expected results discussed in this news release are preliminary estimates only, as Medexus’s financial closing procedures remain subject to completion, and have not been reviewed or audited by the company’s auditors. All such figures are based on information currently available to Medexus management and are subject to change and adjustment as Medexus’s financial results for fiscal Q3 2025 are finalized. Accordingly, final reported results may differ, and may differ materially, from these preliminary estimates, and investors therefore should not place undue reliance on any such preliminary estimates. All such preliminary estimates constitute forward-looking information within the meaning of applicable securities laws, are based on a number of assumptions, and are subject to a number of risks and uncertainties. For more information, see “Forward-looking statements” below. Medexus currently expects to file its financial statements and MD&A for fiscal Q3 2025 after markets close on February 5, 2025.
Medexus expects key operational highlights for fiscal Q3 2025 to include the following –
IXINITY® (US): Continuing slight decline in unit demand, reflecting the continued effect of previously disclosed trends and factors.
Rupall® (Canada): Continuing strong unit demand growth, reflecting successful execution of the company’s initiatives in advance of the expiration of Rupall’s market exclusivity in late January 2025.
Rasuvo® (US) and Metoject® (Canada): Continuing strong unit demand in the face of sustained competition and the continued effect of previously disclosed trends and factors.
Gleolan® (US): Continuing slightly positive trend in US unit demand growth, reflecting the response to the company’s commercialization efforts and successful execution of the company’s commercial plan to date. There nevertheless continue to be disagreements with the licensor regarding the terms of the US Gleolan agreement, which Medexus continues to seek to resolve through mutual negotiation and pursuant to the terms of the US Gleolan agreement, including its dispute resolution process. Medexus is confident that it has performed its obligations under the US Gleolan agreement and, pending resolution of the US Gleolan agreement, whether by mutually acceptable agreement or otherwise in accordance with its existing terms, currently intends to continue commercializing Gleolan in the United States through to at least March 31, 2025 in accordance with and subject to the terms of the US Gleolan agreement. (See also “Risk Factors and Risk Management-Commercial contract disputes” in Medexus’s most recent MD&A.)
Trecondyv® (treosulfan) (Canada): Continuing strong unit demand growth, reflecting successful execution of the company’s initiatives in support of the product, but which does not yet include the effect of the successful completion of the negotiation process with the pan-Canadian Pharmaceutical Alliance and any subsequent decisions by participating government organizations on public reimbursement of Trecondyv for their regions and jurisdictions.
About GRAFAPEX™ (treosulfan) for injection
GRAFAPEX™ (treosulfan) for injection, an alkylating agent, is indicated in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients one year of age and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). GRAFAPEX™ holds Orphan Drug Designation under the Orphan Drug Act, meaning that the product will benefit from a seven-year period of regulatory exclusivity in the FDA-approved indication.
Full prescribing information for GRAFAPEX™ will be available on the Drugs@FDA drug database at www.fda.gov.
Efficacy was evaluated in MC-FludT.14/L Trial II (NCT00822393), a randomized active-controlled trial comparing treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation. Eligible patients included adults 18 to 70 years old with AML or MDS, Karnofsky performance status ≥60%, and age ≥50 years or hematopoietic cell transplantation comorbidity index [HCTCI] score >2. There were 570 patients randomized to treosulfan (n=280) or busulfan (n=290).
The major efficacy outcome measure was overall survival (OS), defined as the time from randomization until death from any cause. The hazard ratio for OS (stratified by donor type and risk group) compared to busulfan was 0.67 (95% CI: 0.51, 0.90) in the randomized population, 0.73 (95% CI: 0.51, 1.06) in patients with AML, and 0.64 (95% CI: 0.40, 1.02) in patients with MDS.
The most common adverse reactions (≥20%) were musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting. Selected Grade 3 or 4 nonhematological laboratory abnormalities were increased GGT (gamma-glutamyl transferase), increased bilirubin, increased ALT (alanine aminotransferase), increased AST (aspartate aminotransferase), and increased creatinine.
The recommended treosulfan dose is 10 g/m2 daily on days -4, -3, and -2 in combination with fludarabine 30 mg/m2 daily on days -6, -5, -4, -3, and -2, and allogeneic hematopoietic stem cell infusion on day 0.
For more information about GRAFAPEX™, including important safety information, see the full prescribing information, which will be available on the Drugs@FDA drug database at www.fda.gov. For more information about the pivotal phase 3 clinical trial of treosulfan conducted by medac GmbH, including its methods, results, and conclusions, and about the publication of the study in the American Journal of Hematology, including a link to the full publication, see Medexus’s June 6, 2022 press release, including the section entitled “About the study”, available on the Investors-News & Events section of Medexus’s corporate website.
GRAFAPEX™ (treosulfan) for injection is approved by the FDA for sale and use in the United States only and is not intended for export outside the United States. Medexus makes no representation that GRAFAPEX™ (treosulfan) for injection is appropriate for, or authorized for sale to or use by, persons who are not located in the United States.
About Medexus
Medexus is a leading specialty pharmaceutical company with a strong North American commercial platform and a growing portfolio of innovative and rare disease treatment solutions. Medexus’s current focus is on the therapeutic areas of oncology, hematology, rheumatology, auto-immune diseases, allergy, and dermatology. For more information about Medexus and its product portfolio, please see the company’s corporate website at www.medexus.com and its filings on SEDAR+ at www.sedarplus.ca.
Victoria Rutherford | Adelaide Capital Tel: 480-625-5772 | Email: victoria@adcap.ca
Forward-Looking statements
Certain statements in this news release contain forward-looking information within the meaning of applicable securities laws, also known and/or referred to as “forward-looking information” or “forward-looking statements.” The words “anticipates”, “believes”, “expects”, “will”, “plans”, “potential”, “prospects”, and similar words, phrases, or expressions are often intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words, phrases, or expressions. Specific forward-looking statements in this news release include, but are not limited to, information contained in statements regarding any of the following: Medexus’s expectations and plans regarding future growth, revenues, and expenses (including in respect of the commercialization of GRAFAPEX™ (treosulfan) for injection and the product-level revenue to be generated from its commercialization in the United States); the potential benefits of GRAFAPEX™ (treosulfan) for injection; the expected timing of any commercial launch of the product in the United States and related expectations regarding GRAFAPEX™ (treosulfan) for injection and the product’s prospects and performance, including in respect of its potential adoption and use in the United States and any related product-level revenue, and including the potential competitive position of the product and anticipated trends and potential challenges in the market in which the product is expected to compete; the expected outcome of Medexus’s and medac’s ongoing evaluation of the milestone amount payable under the US treosulfan agreement; Medexus’s capital allocation strategy, including expectations regarding availability of cash on hand and funds from operations, cash flow generation, and capital allocation and anticipated cash needs, capital requirements, and needs for and ability to secure additional financing (in particular any expectations regarding payment of the regulatory milestone payment that became payable under the company’s GRAFAPEX agreement upon the occurrence of, and which depends on the terms of, the FDA’s approval); and the preliminary estimates of, and any commentary regarding, Medexus’s operating and financial results for the company’s fiscal Q3 2025 (which remain subject to completion of Medexus’s financial closing and other procedures). Finally, forward-looking statements in this news release include statements regarding the occurrence, timing, and expected outcome, and any related consequences for the product and Medexus, of Medexus’s ongoing negotiations and disagreements with the licensor of Medexus’s commercialization rights to Gleolan with respect to the US Gleolan agreement, including any informal and/or formal dispute resolution processes that the parties are currently pursuing and will continue to pursue in future, and otherwise regarding the business relationship of the parties in the United States and Canada. These statements and information are based on Medexus’s current expectations and assumptions, including factors or assumptions that were applied in drawing a conclusion or making a forecast or projection, and including assumptions based on regulatory guidelines, historical trends, current conditions, and expected future developments. In particular, and without limiting the generality of the foregoing, Medexus’s estimate of product-level revenue from commercialization of GRAFAPEX™ (treosulfan) for injection in the United States is based on a number of such factors and assumptions, as described in Medexus’s most recent annual information form and management’s discussion and analysis, and including Medexus’s planned commercial, market access, and medical strategies, the success of which will depend in part on the US regulatory landscape and related dynamics, including potential future changes to each, and can introduce and affect exposure to commercial, legal, and regulatory risk. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. Medexus cautions that, although the assumptions are believed to be reasonable in the circumstances, these risks and uncertainties mean that actual results could differ, and could differ materially, from the expectations contemplated by the forward-looking statements. Material risk factors include, but are not limited to, those set out in Medexus’s materials filed with the Canadian securities regulatory authorities from time to time, including Medexus’s most recent annual information form and management’s discussion and analysis. In addition, specific risks and uncertainties relevant to the content of this news release include, among other things: the uncertainties inherent in research initiatives (including the possibility of unfavorable new data and further analyses of existing data); the risk that data are subject to differing interpretations and assessments by relevant third parties; and whether relevant third parties will be satisfied with the design and methodology of and results from the relevant study, which will depend on many factors, including determinations as to whether the product’s benefits outweigh its known risks and determinations of the product’s efficacy and cost-effectiveness in the context of a given facility (which varies by facility type). Accordingly, undue reliance should not be placed on these forward-looking statements, which are made only as of the date of this news release. Other than as specifically required by law, Medexus undertakes no obligation to update any forward-looking statements to reflect new information, subsequent or otherwise.
Additional notes
Solely for convenience, trademarks and other protected names and marks referred to in this news release sometimes appear without the “®”, “™”, or other similar symbols. Each such reference should be read as though it appears with the relevant symbol. Any such references are not intended to indicate, in any way, that the holder or holders will not assert those rights to the fullest extent under applicable law.
The information in this news release is provided for informational purposes to investors in Medexus securities.
Uniform resource locators, or website addresses, that appear in this news release are intended to be provided as inactive textual references only. Information contained on or accessible through these website addresses is not a part of this news release and is not incorporated by reference into this news release or any of Medexus’s public filings.
Non-GAAP measures
Company management uses, and this news release refers to, financial measures that are not recognized under IFRS and do not have a standard meaning prescribed by generally accepted accounting principles (GAAP) in accordance with IFRS or other financial or accounting authorities (non-GAAP measures). These non-GAAP measures may include “non-GAAP financial measures” and “non-GAAP ratios” (each defined in National Instrument 52-112, Non-GAAP and Other Financial Measures Disclosure). Medexus’s method for calculating these measures may differ from methods used by other companies and therefore these measures are unlikely to be comparable to similarly-designated measures used or presented by other companies.
In particular, management uses Adjusted EBITDA as a measure of Medexus’s performance. EBITDA (earnings before interest, taxes, depreciation, and amortization) and Adjusted EBITDA are non-GAAP financial measures.
An explanation and discussion of each of these non-GAAP measures, including their limitations, is set out under the heading “Preliminary Notes-Non-GAAP measures” in Medexus’s most recent management’s discussion and analysis, and are hereby incorporated by reference. A reconciliation of Adjusted EBITDA to the most directly comparable IFRS measure can be found under the heading “Reconciliation of Adjusted EBITDA to Net Income (Loss)” below.
Reconciliation of Adjusted EBITDA to Net Income (Loss)
The following table is derived from and should be read together with Medexus’s interim condensed consolidated statement of operations for the three- and nine-month periods ended December 31, 2023. This supplementary disclosure is intended to more fully explain disclosures related to Adjusted EBITDA and provides additional information related to Medexus’s operating performance. However, Medexus’s non-GAAP measures have limitations as analytical tools and should not be considered in isolation or as a substitute for analysis of Medexus’s financial information as reported under IFRS.
(Amounts in $ ‘000s)
Three-month periods ended December 31
2023
2022
Net loss
(534)
(1,507)
Add back:
Depreciation and amortization (property, equipment, intangible assets)
1,448
1,515
Interest expense
2,656
3,552
Income tax expense (recovery)
(261)
547
EBITDA
3,309
4,107
Add back:
Share-based compensation
211
436
Transaction-related fees
–
–
Termination benefits
–
372
Foreign exchange loss (gain)
(293)
(338)
Unrealized gain (loss) on fair value of derivatives
Endogenex, Inc., a clinical-stage medical device company, is proud to announce the addition of Krissy Wright as Chief Financial Officer (CFO). In this role, Wright will lead Endogenex’s financial strategy, corporate development initiatives, and operational planning to support the advancement of the company’s clinical initiatives.
“We are thrilled to welcome Krissy to the Endogenex leadership team,” said Stacey Pugh, CEO of Endogenex. “Her extensive experience in financial strategy, corporate development, and leadership in the MedTech industry will be instrumental as we work to achieve our clinical milestones and deliver transformative solutions to type 2 diabetes management.”
Krissy brings experience from Cleerly, an AI-driven heart diagnostics company, where she served as CFO, and from Medtronic, where she held leadership roles including Vice President and General Manager of Brain Modulation and Vice President and Chief Financial Officer for the Neuromodulation Operating Unit. Earlier in her career, she spent 11 years at PricewaterhouseCoopers, specializing in Audit, Assurance, and Transaction Services.
“I am thrilled to be joining Endogenex at such a pivotal moment in its journey,” said Wright. “The company’s novel approach to addressing type 2 diabetes can potentially transform the lives of millions worldwide. I look forward to leveraging my financial expertise to support the team in advancing this groundbreaking therapy and achieving our mission of delivering meaningful, scalable healthcare solutions.”
The ReCET Procedure: ReCET is a novel, endoscopic, outpatient procedure that targets the underlying cellular abnormalities in the duodenum that contribute to the development and progression of type 2 diabetes.
By applying highly controlled, non-thermal pulsed electric fields, the ReCET Procedure is designed to initiate the body’s natural regenerative process to restore proper cellular signaling from the duodenum and improve glycemic control.
The ReCET Procedure is currently being evaluated in global pre-commercialization trials assessing its safety and efficacy in adults with type 2 diabetes who are inadequately controlled despite the use of insulin and non-insulin medications. The ReCET Procedure has received Breakthrough Device Designation from the FDA for the treatment of type 2 diabetes in adult patients inadequately controlled by glucose-lowering medications.
About Endogenex: Endogenex is a privately held, clinical-stage company headquartered in Minneapolis, MN. Endogenex was founded in partnership with Mayo Clinic to develop therapies to improve outcomes for people living with type 2 diabetes. Endogenex focuses on the principle that effective treatment for type 2 diabetes can extend beyond pharmaceutical options, correct metabolic function, and help people regain control of their blood glucose levels.
For more information, please visit our website at www.endogenex.com.
SpectraWAVE, Inc., a medical imaging company focused on improving the treatment and outcomes for patients with coronary artery disease (CAD), today announced the addition of two seasoned executives to its leadership team. Brad Davis has been named Chief Commercial Officer (CCO), and Bill Kelly has been named Chief Financial Officer (CFO).
“The addition of these exceptional leaders to our team represents an exciting inflection point for SpectraWAVE as we enter the new year,” said Eman Namati, Ph.D., Chief Executive Officer of SpectraWAVE. “The response to our recently launched HyperVue Imaging System for optimizing coronary stenting procedures in the catheterization lab has been tremendous. As we plan to expand our commercial footprint and product portfolio, bringing Brad and Bill onto the team is a vital step towards ensuring our success. Their combined expertise in commercialization and financial management will be instrumental in advancing our mission to improve patient outcomes, empower physicians, and create long-term value for our shareholders, building on the momentum of our $50M Series B funding.”
As Chief Commercial Officer, Brad will lead global strategy and commercialization efforts at SpectraWAVE. He brings over 24 years of experience in cardiovascular medical devices, having worked for both large and small companies, including Guidant, Boston Scientific and CSI. Most recently, Brad was CCO at OpSens, where he led the scale-up of a similarly sized commercial team and rapidly grew the business, successfully culminating in an acquisition by Haemonetics. “I am thrilled to join SpectraWAVE at such a pivotal moment in its journey,” said Davis. “The newly launched HyperVue Imaging System provides incredible value to patients, and will serve as a central hub in the cath lab for future enhancements, allowing us to further partner with customers and significantly improve outcomes for patients with coronary artery disease.”
Bill Kelly, newly appointed Chief Financial Officer, will lead SpectraWAVE’s financial strategy, investor relations, and operational efficiency. He joins SpectraWAVE from Vicarious Surgical, where he served as CFO and successfully took the company public, raising over $275 million through initial and follow-on public offerings. Prior to his tenure at Vicarious Surgical, Bill was CFO at Exosome Diagnostics, where he played an instrumental role in raising capital and launching commercial operations, ultimately leading to the company’s acquisition by BioTechne. “SpectraWAVE has built a powerful imaging platform in a critical area of healthcare – management of coronary artery disease,” noted Kelly. “I’m excited to join the team during an exciting period of growth and collaborate across the organization to ensure we have the resources and infrastructure to meet our short- and long-term objectives.”
Intravascular imaging is an essential tool to optimize coronary stenting procedures, providing key insights into plaque morphology, plaque modification decisions, stent and balloon sizing and landing zone selection, confirmation of treatment optimization, and future adverse event risk. Prior intravascular imaging technologies offer compromises in image resolution, image depth, and ease of use. HyperVueTM is the first intravascular imaging technology to combine two important imaging technologies, DeepOCT and NIRS, while optimizing for image quality and procedural efficiency in the cath lab, including no-flush catheter prep, fast and long pullbacks designed to reduce and remove the use of contrast, and the most comprehensive AI-driven workflow and image analysis offering.
About SpectraWAVE, Inc.
SpectraWAVE, located in Bedford, Mass., is a privately held medical device company founded in 2017 to provide unrivaled optical and computational insights to improve the treatment and outcomes for patients with coronary artery disease (CAD). CAD, the buildup of plaque in the wall of the arteries that supply blood to the heart, affects 20.5 million adults aged 20 and older. In CAD patients that undergo percutaneous stent placement, it is estimated that one in five patients experience adverse events within two years. SpectraWAVE’s flagship HyperVueTM Imaging System combines next generation DeepOCTTM images and near infrared spectroscopy (NIRS) with workflows optimized for the cardiac catheterization lab, and serves as a central hub for future enhancements that will continue to empower interventionalists in their treatment decision making and optimization. The HyperVue Imaging System is intended for the imaging of coronary arteries and is indicated in patients who are candidates for transluminal interventional procedures. The NIRS capability of the system is intended for the identification of patients and plaques at increased risk of major adverse cardiac events. For more information and complete indications for use, please visit www.spectrawave.com.
Lumira Ventures, a leading North American life sciences investment firm dedicated to advancing healthcare innovation and enhancing patient outcomes, is pleased to announce the promotion of Suman Rao, Ph.D., to the role of Principal. This well-deserved promotion acknowledges her significant contributions to the firm’s success and her steadfast commitment to fostering innovative healthcare solutions.
Dr. Rao joined Lumira Ventures in 2021 as an Associate and was promoted to Senior Associate in 2024, as she built out her skills and experience in all dimensions of the venture capital value chain. Her deep understanding of industry sectors like oncology and neuroscience coupled with her ability to foster strong relationships with entrepreneurs, co-investors, and industry stakeholders has made her a sought-after team member. Dr. Rao has been deeply involved with the firm’s investments in Arclight Therapeutics, Congruence Therapeutics, and Damona Pharmaceuticals (as a board observer). Her contributions extend to being actively engaged in a number of the firm’s strategic initiatives, including the Angelini Lumira Biosciences Fund and currently co-leading the Cancer Breakthrough Fund, a first-of-its-kind collaboration in Canada between a philanthropic organization (Terry Fox Foundation) and a VC firm with the goal to bring more capital to the mission of finding cures for cancers.
“We are delighted to elevate Suman to the role of Principal,” said Peter van der Velden, Managing General Partner at Lumira Ventures. “Her growth at the firm and ability to take on more responsibility reflects our commitment to growing talent in-house and her dedication to developing the skills, expertise, and relationships that are the foundation of being a great VC.”
“I am thrilled to be stepping into the role of Principal and grateful for the trust and support of my partners and colleagues,” said Dr. Rao. “Together with an incredibly talented team, I look forward to supporting passionate entrepreneurs tackling some of medicine’s biggest challenges today and building transformative companies.”
Dr. Rao earned her Ph.D. in Experimental Medicine and her Bachelor’s degree in Biochemistry from McGill University. Before joining Lumira Ventures, she completed a prestigious postdoctoral fellowship at the Dana-Farber Cancer Institute and Harvard Medical School. Dr. Rao also brings valuable experience from her time as a management consultant at L.E.K Consulting, where she provided strategic advice to biotech and pharma clients, further honing her expertise in the life sciences sector.
About Lumira Ventures Lumira Ventures is a leading multi-stage life sciences investment firm operating across Toronto, Montréal, Vancouver, and Boston. As Canada’s largest and most active dedicated life sciences venture capital investor, Lumira invests in first-in-class innovations in “secondary” geographies, partnering with mission-driven entrepreneurs to develop transformative therapeutics products and medical devices while generating strong financial returns and societal impact.
With a proven track record, Lumira Ventures has consistently delivered both financial success and transformative medical advancements. With portfolio companies having introduced over 50 biomedical innovations, improving the lives of over 1 billion patients worldwide and generating more than $70B in cumulative revenue.
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, has secured Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CNP-104 for the treatment of primary biliary cholangitis (PBC).
“Receiving Orphan Drug Designation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “This designation follows our presentation of positive topline data from the Phase 2a clinical trial of CNP-104 in PBC at The Liver Meeting® 2024. Notably, in addition to demonstrating favorable T cell responses among treated participants, CNP-104 slowed disease progression, as evidenced by a statistically significant reduction in liver stiffness measured by FibroScan, by day 120 of the study period. We are now collaborating with our distinguished clinical advisors and key opinion leaders to advance CNP-104 to the next phase of clinical development.”
The FDA grants ODD status to encourage the development of treatments for rare diseases affecting less than 200,000 individuals in the United States. This designation qualifies sponsors for various incentives, including tax credits for clinical trials, exemption from user fees, and the potential for seven years of market exclusivity following approval. CNP-104 additionally received Fast Track Designation from the FDA in January 2022, which would make it eligible for Accelerated Approval and Priority Review if specific criteria are met.
About CNP-104:
CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic activated PDC-E2 T-cells that drive inflammation in bile ducts, leading to improvement in clinical outcomes of liver health.
About PBC:
PBC is a chronic, life-threatening autoimmune disease of the liver that disproportionately affects women, and is a leading cause of liver transplants in this population. PBC is characterized by impaired bile flow (cholestasis) and the accumulation of toxic bile acids in the liver, which can lead to fibrosis, cirrhosis and ultimately liver failure, necessitating a transplant. Symptoms such as fatigue and pruritus (severe itching) significantly affect patients’ quality of life. While the exact cause of PBC is unknown, an autoimmune response to the E2 component of the pyruvate dehydrogenase complex (PDC), is thought to be highly associated with the development of the disease.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is currently enrolling patients in a Phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. Additionally, COUR is developing an undisclosed pre-clinical stage program in collaboration with Genentech.
XyloCor Therapeutics, Inc., (“XyloCor”), a clinical stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced the completion of a $67.5 million Series B financing.
New investment will support a randomized, double-blind Phase 2b clinical trial (EXACT-2) of XC001 (encoberminogene rezmadenovec), in refractory angina, using a new non-surgical method of endocardial administration via a novel injection catheter. The financing will also fund a second randomized, double-blind Phase 2 trial of XC001 as an adjunctive treatment to coronary artery bypass graft surgery (CABG). XC001 offers a new therapeutic approach for debilitating and chronic conditions that impact over one million people in the United States who have no treatment options.
New investor Jeito Capital, a global leading private equity fund, led the Series B financing round which also included existing institutional investors EQT, Fountain Healthcare Partners, and Lumira Ventures. Rachel Mears, Partner at Jeito Capital, will join the XyloCor Board of Directors.
“We are delighted to have Jeito Capital join our strong investor syndicate and Board of Directors,” said Al Gianchetti, president and chief executive officer of XyloCor Therapeutics. “The support of this prominent group of life sciences investors is recognition of the progress we have made and confidence in our ability to reach important milestones in the path ahead. With this financing, we can accelerate our clinical development of XC001, completing two phase 2 clinical trials, and achieve our mission to help people with cardiovascular disease who have no treatment options.”
XyloCor is pioneering the application of one-time gene therapy to address significant unmet treatment needs among underserved patients with cardiovascular disease. In its initial target indication in refractory angina, XC001 has demonstrated potential to transform outcomes for patients who have exhausted available treatment options and have a debilitating quality of life. Positive results from the recently published Phase 1/2 clinical trial (EXACT-1) demonstrate the disease-modifying potential of XC001 to relieve chest pain in patients with refractory angina by reducing ischemic burden, as published in Circulation: Cardiovascular Interventions.
Based on the foundation of efficacy and safety data for XC001 demonstrated in EXACT-1, XyloCor plans to launch a randomized, double-blind Phase 2b in refractory angina in 2025 to further build upon the clinically-meaningful evidence generated to-date. XyloCor intends to deploy a catheter‑based endocardial delivery of XC001 in the Phase 2b EXACT-2 study, eliminating the need for surgical administration in this population.
XyloCor also aims to initiate a second Phase 2 trial of XC001 in 2025 as an adjunctive therapy to augment the effectiveness of CABG: a procedure used to treat coronary artery disease. CABG is generally recommended when there are significant blockages in the major coronary arteries with the objective to improve oxygen-rich blood flow, resulting in improvement in cardiovascular disease symptoms and quality of life, and reduction in future cardiac events. There are approximately 400,000 CABG procedures performed annually in the United States, in which an estimated one-third of procedures result in incomplete coronary revascularization, which can result in increased mortality, hospitalizations, repeat revascularizations, and angina symptoms. Administering XC001 during the CABG procedure is intended to promote the growth of new blood vessels in the areas of the heart not treated by the bypass grafts and therefore reduce symptoms and improve outcomes beyond the bypass alone. XyloCor plans to dose the first patient in the Phase 2 study by year end 2025.
“We are thrilled to support XyloCor as it advances its clinical trials to evaluate XC001 as a potential treatment for patients struggling with the burden of cardiovascular disease,” said Rachel Mears, Partner at Jeito Capital. “The company has strong support from an experienced leadership team, world-class cardiologists and scientists and has made impressive achievements in a short time in advancing its novel gene therapy approach. We look forward to collaborating with the company as it progresses to the next steps in its clinical program.”
About Jeito Capital
Jeito Capital is a global leading Private Equity fund with a patient benefit driven approach that finances and accelerates the development and growth of ground-breaking medical innovation. Jeito empowers and supports managers through its expert, integrated, multi-talented team and through the investment of significant capital to ensure the growth of companies, building market leaders in their respective therapeutic areas with accelerated patients’ access globally, especially in Europe and the United States. Jeito Capital has €534 million under management and a rapidly growing portfolio of investments. Jeito Capital is based in Paris with a presence in Europe and the United States. For more information, please visit www.jeito.life or follow us on LinkedIn or X.
About XC001
XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with ischemic heart disease may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.
About XyloCor
XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with ischemic heart disease for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.
Lumira Ventures is proud to introduce the newest cohort joining our Venture Innovation Program (VIP) for the 2024-2025 term. The VIP program offers Ph.D., MBA, and MD students across Canada a unique opportunity to gain hands-on experience in life sciences venture capital and startups, nurturing the next generation of leaders, and contributing to the future of healthcare innovation.
We welcome Xavier Lee from the University of Toronto and Sam Fisher from Dalhousie University. These talented individuals bring diverse backgrounds and expertise that align with Lumira’s mission to drive innovation in the life sciences sector. We are excited to welcome them to the Lumira Ventures family. Their fresh perspectives and innovative ideas will undoubtedly contribute to our team and the broader life sciences community while furthering their careers in life sciences innovation and venture capital.
Meet Our Fellows
Xavier Lee, University of Toronto
Xavier is a 5th year Ph.D. candidate in Physiology at the University of Toronto. His research focuses on right-ventricular heart disease, employing a multidisciplinary approach that combines computational, physiological, and molecular methodologies.
Beyond his academic pursuits, Xavier has demonstrated strong leadership skills through his involvement in various University of Toronto organizations, where he has worked to foster connections among graduate students and industry professionals. His experience with two consulting firms, providing strategic and operational solutions to healthcare and life sciences clients, adds valuable industry insight to his profile.
Sam Fisher, Dalhousie University
Sam is completing his Ph.D. in Chemistry at Dalhousie University, where his research centers on enhancing sustainability in small molecule pharmaceutical synthesis. His work involves collaborations with leading life science and pharmaceutical companies, bridging academic research with industry needs. Prior to his doctoral studies, Sam held a senior position at Breathe Biomedical, gaining experience in investor relations, financial modeling, and intellectual property management.
At Dalhousie, Sam has been a champion for equity, diversity, and inclusion in the sciences, while also engaging in STEM outreach programs and mentoring at-risk youth.
About the Lumira Venture Innovation Program
The Lumira VIP is a 6–12-month immersive experience tailored to fit alongside academic studies. Participants will engage in various aspects of life sciences venture innovation and investment, working closely with seasoned industry professionals. This unique program provides aspiring leaders in the life sciences with:
Comprehensive exposure to the full lifecycle of venture innovation and investment.
Hands-on experience with projects spanning science, business strategy, and operations.
Flexible work arrangements with opportunities for remote and in-person collaboration.
Financial support, including a monthly stipend for participants.
At Lumira Ventures, we’re dedicated to fostering innovation and empowering the next generation of leaders in Canada’s thriving life sciences ecosystem.
About Lumira Ventures
Lumira Ventures is a leading multi-stage life sciences investment firm operating across Toronto, Montréal, Vancouver, and Boston. As Canada’s largest and most active dedicated life sciences venture capital investor, Lumira invests in first-in-class innovations in “secondary” geographies, partnering with mission-driven entrepreneurs to develop transformative therapeutics products and medical devices while generating strong financial returns and societal impact.
With a proven track record, Lumira Ventures has consistently delivered both financial success and transformative medical advancements. Our portfolio companies have introduced over 50 biomedical innovations, improving the lives of over 1 billion patients worldwide and generating more than $70 billion in cumulative revenue.
Congruence Therapeutics today announced that it has entered into a research collaboration agreement with Ono Pharmaceutical Co., Ltd. (Ono) to discover novel drug candidates leveraging Congruence’s proprietary drug discovery platform, Revenir™. Congruence will apply its technology and expertise toward multiple cancer protein targets. Once the discovery effort advances to a prespecified stage, Ono has the option to acquire exclusive worldwide rights to further develop and commercialize small molecule correctors generated during the collaboration.
“We believe that this collaboration with Congruence may help generating novel small molecule correctors for validated targets in the oncology area by leveraging their own technologies in protein dynamics and computational biology, leading to our development pipeline,” said Seishi Katsumata, PhD, Corporate Officer / Executive Director, Discovery & Research of Ono. “We will be committed to delivering innovative new drugs to cancer patients as soon as possible.”
The collaboration will leverage Congruence’s purpose-built computational drug discovery engine called Revenir™, which captures the biophysical changes caused by mutations in proteins. By examining surface features and numerous biophysical descriptors of both the mutated and wild-type proteins, Congruence can derive novel insights regarding protein defects and how to correct them. Congruence’s current pipeline consists of both first-in-class and best-in-class potential therapeutic candidates that address significant unmet medical need in a number of high value indications.
“Congruence is thrilled to partner with Ono, which has established itself as a global leader in drug development, particularly in the oncology space. We believe that our Revenir™ platform and capabilities in protein dynamics will accelerate the discovery of novel therapies for compelling targets of interest to both companies,” said Sharath Hegde PhD, Chief Scientific Officer of Congruence.
Under the terms of the collaboration, Congruence will receive an undisclosed upfront payment and is eligible to receive milestone payments upon the achievement of certain discovery, development, approval and sales events – as well as tiered royalties based on annual net sales of related products. Ono will additionally reimburse Congruence for the research costs it incurs in connection with the collaboration. Ono has the option to take an exclusive worldwide license to development candidates discovered under the collaboration.
About Revenir™ Drug Discovery Platform
Revenir™, Congruence’s proprietary computational drug discovery platform, captures the dynamic biophysical changes caused by mutations in proteins, offering unique insights into protein defects and their correction. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir™ predicts small molecule induced correction of the underlying defect.
About Congruence Therapeutics
Congruence is a computationally-driven biotechnology company building a unique pipeline of transformative small molecule correctors rationally designed to rescue aberrant protein function. Our proprietary scalable platform, Revenir™, captures the biophysical features of proteins across their conformational ensembles, in order to identify novel allosteric and cryptic pockets which are virtually screened to generate novel chemical matter.
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced a strategic collaboration and licensing agreement with Genentech, a member of the Roche Group, for the development and commercialization of COUR’s proprietary tolerogenic nanoparticle treatments for an autoimmune disease.
“Genentech has a long history of innovation, and we are excited to collaborate on the development of disease-modifying therapies leveraging COUR’s proprietary tolerance platform for the treatment of an autoimmune disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “We see Genentech as an ideal partner with world-class scientific expertise and together have an opportunity to build upon COUR’s clinical success to date in autoimmune diseases. Through this partnership, we believe we have the potential to have a significant impact on an important patient community.”
“We have a long and successful legacy of advancing innovative science and developing transformative medicines for people diagnosed with autoimmune diseases,” said Boris L. Zaïtra, Head of Roche Corporate Business Development. “Collaborating with innovative partners such as COUR supports our rigorous commitment to pioneering science in pursuit of advances in research, such as novel tolerogenic nanoparticle treatments, to deliver the most impactful medicines for patients with significant unmet needs.”
Under the terms of the agreement, COUR is eligible for upfront and near-term milestones of $40 million, plus additional development, commercial and net sales milestone payments that could exceed $900 million, as well as tiered royalties on net sales. Under the collaboration agreement with Genentech, COUR is responsible for the preclinical development and technical transfer of manufacturing. Genentech will be responsible for clinical development, regulatory filing, and commercialization.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.
KisoJi Biotechnology Inc., a company focused on the discovery and development of transformative antibody therapeutics, announces that it has raised $41 million in equity, including the conversion of previously funded convertible debentures. These funds will be used to advance its lead asset, KJ-103, the first potent naked antibody against TROP2, into the clinic to treat solid tumours. Funds will also be used to deploy its cutting-edge antibody discovery platform towards new multi-specific therapeutic antibody drugs in cardiometabolic disease, as well as immunology and inflammation. The financing was led by Investissement Quebec and Lumira Ventures with participation by Fonds de solidarite FTQ, adMare BioInnovations, and Remiges Ventures.
As previously announced, KisoJi has established a clinical development partnership with Cancer Research UK to bring KisoJi’s lead asset, KJ-103, into a first-in-human clinical trial of approximately 100 patients. KJ-103 is highly differentiated from existing TROP2-targeting drugs on the market or in development, which are antibody drug conjugates (ADCs). KJ-103 does not require a cytotoxic payload but instead functions by recruiting immune cells to kill tumour cells.
The financing will also support the deployment of KisoJi’s cutting-edge antibody discovery platform, including the first of its kind antibody paratope map (KisoSeekTM) that effectively visualizes an immune response against a given target. The Company’s sampling tools allow it to maximize paratope diversity in a directed iterative strategy to focus on distinct antibodies and uncover novel biology, including functional GPCR antibodies.
KisoSeekTM is more efficient and provides greater insights than traditional antibody discovery methods and can integrate seamlessly into existing workflows. All existing data, whether positive or negative, can be used to augment a target specific paratope to supercharge antibody discovery instead of relying on traditional methods of random screening.
KisoJi has also developed a suite of camelid transgenic mice (KisoMouse®) that generate single domain antibodies to continue populating the world’s largest single domain antibody database created by KisoJi over the past 8 years. The paratope map and antibody database can be used to generate first-in-class and best-in-class multi-specific antibodies using KisoJi’s modular multi-specific antibody format (KisoBodyTM). The KisoBodyTM is easily customizable and manufacturable with tri-specific antibody titers at greater than 6 g/L in commercial production cell lines.
Bicha Ngo, President and CEO, Investissement Québec, said: “By contributing to KisoJi Biotechnology’s financing round, Investissement Québec is reaffirming its commitment to supporting innovative companies in the life sciences industry, a key sector for the Québec economy. This funding will allow KisoJi Biotechnology to hit critical milestones in its operations, from completing preclinical activities to launching clinical trials, notably through a landmark agreement with Cancer Research UK, one of the world’s largest funders of cancer research, all the while helping move innovations from the R&D stage to the marketplace.”
Daniel Hétu, Managing Director, Lumira Ventures, said: “KisoJi has built a truly differentiated, AI enabled and comprehensive antibody discovery platform leveraging the deep expertise of their team that spans transgenic mice, single-domain antibodies and AI. Lumira is proud to have supported the development of KisoJi’s innovative platform from its inception and we look forward to continuing to work with the company and our co-investors as KisoJi transitions its propriety programs to the clinical stage.
Maxime Pesant, Vice-President Private Equity and Impact Investing | Life Sciences, Fonds de solidarité FTQ, said: “The Fonds de solidarité FTQ is proud to reiterate its commitment to KisoJi, an innovative company of which we have been partners from the very beginning alongside Lumira. Our strategic investments in the sector help propel high-potential biotechs to new heights. With major expertise in the life sciences field and with our long-term presence, the Fund continues to support high-impact companies like KisoJi, paving the way for groundbreaking innovations and significant medical advancements.”
David Young, co-founder and CEO of KisoJi said: “Through the support of our new and existing investors, this financing will enable KisoJi to continue its development of pipeline assets in oncology, cardiometabolic and immunology indications using our cutting-edge platform and AI tools.”
Evolution Venture Partners acted as the exclusive strategic advisor to KisoJi in connection with the financing.
About KisoJi Biotechnology KisoJi is a Canadian biotechnology company that deploys the latest scientific and AI tools to conduct therapeutic antibody discovery in a fundamentally new way. KisoJi has developed a multi-species transgenic mouse to generate highly diverse single domain antibodies, as well as a modular multi-specific antibody scaffold with high stability and productivity. Most recently, KisoJi has used advanced AI tools to visualise the universe of all antibodies against a target in order to uncover novel biology and new therapeutic antibody capabilities for its partners and its own pipeline.
About KJ-103 KJ-103 is a single domain antibody that binds to a unique epitope on TROP2. It acts via effector cell mechanisms, including the activation of macrophages, to kill tumour cells. KJ-103 has been shown to have significant anti-tumour potency with no evidence of toxicity or resistance across a number of preclinical solid tumour models. KJ-103 was humanised by LifeArc.
KisoJi Biotechnology Contact: Investors and Media Argot Partners Sam Martin/Cameron Willis KisoJi@argotpartners.com +1 212 600 1902
Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced the acquisition of a portfolio of novel nucleoside analogs from PrimeFour Therapeutics, Inc., a biotechnology company focused on cancer chemotherapeutic agents.
The portfolio of novel nucleosides consists of a chemically diverse library of unique analogs. The most advanced lead compounds are nucleosides that cause “synthetic lethality” in cancers characterized by distinct tumor-defined genetic signatures, allowing them to be precisely targeted to the most susceptible cancers with reduced toxicity. These compounds can be targeted towards cancers with newly discovered genetic biomarkers that are present in pancreatic and certain other solid tumors as well as in hematologic cancers, allowing for personalized therapy in those patients most likely to respond to treatment.
“This transaction allows us to build upon our leadership in developing first-in-class and best-inclass nucleoside reverse transcriptase inhibitors for neurodegenerative and autoimmune diseases, including ALS, PSP and Alzheimer’s disease. We are excited to expand our pipeline to include this portfolio of potent nucleoside chemotherapeutics with significant therapeutic potential against a variety of cancers,” said Eckard Weber, M.D., founder and Chief Innovation Officer of Transposon. “We look forward to advancing the lead compounds into preclinical development to target DNA-damage repair deficient cancers, such as pancreatic and colorectal cancers.”
About Transposon Transposon Therapeutics, Inc.
is a clinical-stage biopharmaceutical company developing a platform of novel nucleoside reverse transcriptase inhibitors for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. Transposon is advancing TPN-101 into Phase 3 registration studies for the treatment amyotrophic lateral sclerosis (ALS) and progressive supranuclear palsy (PSP), and a Phase 2 proof-of-concept study in Alzheimer’s disease. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications
About PrimeFour
PrimeFour discovered and developed a class of novel nucleoside analogs that are selectively incorporated into cancer cells with biomarker-identifiable DNA damage repair deficiencies and that, once incorporated into some cancer cells, cause irreversible DNA damage and cancer cell death.
AmacaThera today announced results from its Phase 1 clinical trial of AMT-143, an innovative, non-opioid anesthetic, formulated with the company’s patented AmacaGel™ platform, demonstrating sustained anesthetic release out to 14 days. AMT-143 is intended to deliver extended, localized pain relief without the need for opioids, significantly reducing the risks associated with opioid addiction and side effects in order to improve patient outcomes post-surgery.
“We are excited by the promise of these results as they show a substantial differentiation above and beyond the currently available products and herald a next generation of post-surgical pain relief alternatives,” said Dr. Mike Cooke, Chief Executive Officer, and Co-Founder of AmacaThera, “We look forward to advancing this important product into a Phase 2 study.”
The recent clinical data has strengthened support for AmacaThera with lead investor of AmacaThera’s recently completed Series A extension Paul Austin saying “The progress shown in the Phase 1 study demonstrates the tremendous potential of AMT-143 to transform post-operative pain management. Based on these strong results, we are increasing our investment to accelerate its continuing development.”
The urgent need for non-opioid, long-acting anesthetics stems from the ongoing opioid crisis, where opioid-based pain management serves as a gateway to opioid abuse. While effective for acute pain relief, opioids can result in addiction, overdose, and adverse side effects, creating a pressing need for safer alternatives. Non-opioid long-acting anesthetics offer a viable solution by providing effective pain relief without the risk of dependency or the severe side effects commonly associated with opioids.
Dr. Molly Shoichet, Chief Scientific Officer and Co-Founder of AmacaThera: “The Phase 1 clinical data, in combination with our optimized release kinetics, cost effective manufacturing, and ease of application comprise the solution physicians want for their patients.”
About AmacaThera
AmacaThera is a venture backed clinical stage biotechnology company supported by institutional investors including; Lumira Ventures, BDC Capital’s Women in Technology Venture Fund, Inveready, StandUp Ventures, MaRS IAF, and Changrong Capital.
AmacaThera is specializing in the development of advanced sustained release hydrogel formulations to solve key therapeutic delivery challenges with both off-patent and proprietary payloads. AmacaThera is focused on developing an internal pipeline of pain management and oncology assets; and partnering to solve key delivery challenges with advanced proprietary therapeutic modalities.
AmacaThera’s platform technology, AmacaGelTM, is a fast-gelling physical hydrogel blend of two well-established polymers. It has been designed to liquify under shear force and to be delivered by a conventional syringe, thereby rapidly forming a depot as it warms to body temperature. AMT-143, the platform’s lead asset, is a slow-release non-opioid, local anesthetic leverages the AmacaGelTM platform to provide long-acting, post-operative pain relief.
With the global market for non-opioid pain management expected to exceed $96 Billion by 20341. AmacaThera will address a critical gap in post-operative care with AMT-143.
With strong early data, AmacaThera is actively seeking strategic partners to expand the clinical program and accelerate market entry for AMT-143 and related products.
Thryv Therapeutics Inc., a clinical-stage biotechnology company pioneering novel therapies for cardiovascular diseases, is pleased to announce the appointment of Amy Sehnert, MD, as Chief Medical Officer (CMO). Dr. Sehnert will lead the clinical development and strategy for Thryv Therapeutics’ expanding cardiometabolic product portfolio targeting serum glucocorticoid inducible kinase 1 (SGK1) – a kinase implicated in adverse electrical and structural remodeling of the heart.
Dr. Sehnert is a pediatric cardiologist with 20 years of experience as a serial and founding medical lead at innovative biotech companies introducing precision diagnostics and therapeutics to the clinic. Dr. Sehnert joins Thryv Therapeutics from Bristol Myers Squibb (BMS), where she held leadership roles of increasing responsibility and most recently served as Vice President and Global Program Lead for cardiovascular assets transitioning from early to late development. Prior to her time at BMS, Dr. Sehnert served as Vice President, Clinical Science at MyoKardia, Inc., where she played a pivotal role in the development and approval of Camzyos® – a groundbreaking treatment for hypertrophic cardiomyopathy (HCM) – contributing to the $14 billion acquisition of MyoKardia by BMS in 2020.
“I am thrilled to join Thryv Therapeutics at such an important time in the company’s journey,” said Dr. Sehnert. “Thryv Therapeutics’ cutting-edge approach to cardiovascular diseases aligns with my passion for advancing precision medicine, and I look forward to working with the team to deliver transformative therapies to patients worldwide.”
Dr. Sehnert has also made notable contributions in other fields of medicine, such as maternal-fetal health, oncology, and genomics, leading early efforts in non-invasive prenatal testing (NIPT) and cell-free DNA applications now in wide clinical use. She holds a Doctor of Medicine degree from the University of Minnesota, completed Pediatrics Residency at University of Colorado, and Fellowship in Pediatric Cardiology at UCSF followed by faculty appointments at UCSF and Stanford University. In 2022 she was recognized as one of the Most Influential Women in Business by the San Francisco Business Times and among the Top 25 Healthcare Technology Leaders of San Francisco. She has co-authored over 60 peer-reviewed publications and has been involved in numerous international collaborations, making her an internationally recognized expert in her field.
“On behalf of Thryv Therapeutics, I am excited to welcome Amy to the team,” said Paul F. Truex, CEO of Thryv Therapeutics. “Dr. Sehnert brings a wealth of medical and clinical development knowledge in the field of cardiometabolic and genetic diseases. Her extensive experience in designing and executing late-stage clinical trials for genetic and broader cardiovascular diseases and leadership qualities will be invaluable as we enter the next phase of clinical development of our SGK1 portfolio.”
Dr. Philip Sager, Thryv Therapeutics’ founding Chief Medical Officer, will continue to provide strategic clinical and regulatory guidance as the company’s portfolio advances into later stage clinical studies.
“Philip and his collaborators originally developed unique insights into the clinical potential for SGK1 in various cardiometabolic diseases and in particular, Long QT Syndrome. His tireless leadership and relentless passion for exploring these initial ideas advanced Thryv Therapeutics’ portfolio to its current stage,” said Paul F. Truex. “We’re thankful for his continued, on-going contributions in his new role to all of our clinical programs and we are inspired by his commitment to finding treatments for these patient communities.”
About Thryv Therapeutics Inc.
Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop potent and highly selective serum glucocorticoid inducible kinase 1 (SGK1) inhibitors to treat heart failure, atrial fibrillation, Long QT Syndrome and other cardiometabolic and cardiorenal diseases. Numerous animal models have demonstrated that activation of SGK1 leads to inappropriate electrical and structural remodeling of the heart contributing to adverse outcomes in heart failure, atrial fibrillation, and genetic channelopathies, such as Long QT Syndrome. For more information, please visit www.thryvtrx.com.
HistoSonics, the developer and manufacturer of the Edison® Histotripsy System, announced today the first patients were enrolled in their novel prospective master protocol study, called BOOMBOX, designed to evaluate the real-world use of histotripsy for treatment of liver tumors. BOOMBOX is a comprehensive, post-market master study that will provide important data for up to 5,000 patients treated across a growing global base of multi-disciplinary histotripsy providers. This master study design is unique in the medical device industry, as it captures treatment data across diverse tumor types and disease stages in patients with varying interventional needs, and amongst multiple clinical specialties.
“Traditionally, this complexity would require multiple individual study protocols over a prolonged period of time, delaying access to histotripsy for patients in need,” said Amanda Cafaro, VP of Clinical Affairs, HistoSonics. “We designed this post-market master protocol study and its accompanying sub-studies to accelerate our understanding of a wide range of clinically relevant research questions, expediting access to this innovative therapy.”
The primary endpoint for BOOMBOX will examine histotripsy’s capability as a versatile tool and the subsequent sub-studies will evaluate additional endpoints to demonstrate increasingly specific treatment goals: as a monotherapy, a combination therapy, for curative intent as well as priming for additional treatments. Enrollment has begun for the BOOMBOX study across multiple US histotripsy clinical treatment centers and the company expects participation from both US and global histotripsy sites.
“Histotripsy represents a novel treatment for my patients suffering with liver tumors and has already demonstrated an enhanced benefit-risk profile in a clinically meaningful way,” said Juan P. Rocca, M.D., MHA, surgical director for the New York-Presbyterian and Weill Cornell Medicine Liver Cancer Program, one of the centers around the country to offer histotripsy. “By participating in the BOOMBOX study, we are engaging in practical, real-world research that will drive the advancement of this innovative platform and explore new ways to utilize histotripsy in the future. Participating in BOOMBOX is exactly the type of practical research that will continue to advance this groundbreaking new platform and help us find new and innovative ways to use histotripsy going forward.”
“The depth and breadth of data we will capture as part of the BOOMBOX study will provide us and our histotripsy program partners with insight on how histotripsy may impact patients across many diseases and patient conditions. BOOMBOX is our long-term commitment to learning how histotripsy can best improve the lives of patients suffering from liver tumors and the debilitating symptoms these patients face,” said Joe Herman, MD, VP of Medical Affairs, HistoSonics.
The BOOMBOX study begins HistoSonics’ post-market evidence generation efforts in the liver after the recent publication of the company’s pivotal clinical #HOPE4LIVER trials which were submitted to the US Food and Drug Administration (FDA) as part of HistoSonics market access submission resulting in FDA De Novo grant in October 2023 for the non-invasive destruction of liver tumors using histotripsy. In addition to the BOOMBOX: Master Study, HistoSonics has established an ongoing Investigator Sponsored Research program designed to support independent research with histotripsy providers through grants, advancing the safe and effective use of histotripsy.
The Edison System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any disease including, but not limited to, cancer or evaluated any specific cancer outcomes (such as local tumor progression, 5-year survival or overall survival). The System should only be used by physicians who have completed training performed by HistoSonics, and its use guided by the clinical judgment of an appropriately trained physician. Refer to the device Instructions for Use for a complete list of warnings, precautions, and a summary of clinical trial results, including reported adverse events.
About HistoSonics
HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.
Nocion Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions”, that selectively silence actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain, today announced that the first patient has been dosed in the Phase 2b ASPIRE clinical trial of taplucainium for the treatment of chronic cough.
ASPIRE is a Phase 2b, randomized, double-blind, placebo-controlled study investigating the efficacy, safety, and tolerability of Taplucainium Inhalation Powder (NOC-110) once daily in adults with refractory or unexplained chronic cough. Approximately 325 patients will be enrolled in the study at over 100 sites across the US, Canada, UK and Europe.
“Dosing of the first patient in the ASPIRE clinical study is a significant milestone for Nocion,” said Rick Batycky, CEO of Nocion Therapeutics. “Taplucainium represents a novel approach to the treatment of chronic cough with the potential to deliver enhanced efficacy and reduced side effects to more than 8 million patients per year just in the United States.”
ABOUT TAPLUCAINIUM Taplucainium (formerly NTX-1175) is a proprietary molecule in the novel class of charged sodium channel blockers that allows for specific silencing of activated/inflamed nociceptors while having minimal local off-target effects or systemic exposure. Unlike other investigative cough therapies, such as P2X3 antagonists, which target a specific large pore channel, taplucainium is formulated into a dry powder for inhalation, and once inhaled, gains access to the pulmonary nociceptors through any open large pore channel including P2X, TRPV and TRPA channels whereupon it inhibits the sodium channels responsible for initiating the pathological cough response. The broader mechanism of taplucainium should translate into an efficacious, long-lasting, antitussive that is fast acting in a number of cough conditions. Combined with good preliminary safety and efficacy data from earlier stage clinical work, this forms the basis for its use in cough indications beyond chronic cough. For more information, visit https://www.aspirechroniccoughstudy.com/#!/
ABOUT NOCION Nocion Therapeutics is a biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. Venture investors in Nocion include Arkin Bio Capital, Canaan Partners, F-Prime Capital, Lumira Ventures, Mass General Brigham Ventures, Mission BioCapital, Monograph Capital, Morningside and Osage University Partners. For more information, visit: www.nociontx.com.
Thryv Therapeutics Inc., a biotechnology company pioneering treatments for genetic and cardiometabolic conditions, including congenital Long QT Syndrome, heart failure, and atrial fibrillation, has completed phase 1 dosing of its second SGK1 inhibitor. This study was a first-in-human, randomized, double-blind, placebo-controlled, single and multiple ascending dose study to assess the safety, tolerability, pharmacokinetics, and food effect of THRV-1268 in healthy adult subjects. The completion of dosing marks a significant milestone in the development of THRV-1268, laying the groundwork for future clinical investigations in heart failure and atrial fibrillation.
“Completion of this first-in-human study is the initial clinical step with THRV-1268, providing a path towards addressing cardiometabolic diseases where both structural and electrical remodeling lead to adverse CV outcomes in patients with atrial fibrillation and heart failure,” stated Philip Sager, MD, Chief Medical Officer of Thryv Therapeutics. “Building on our initial clinical work with LQT-1213 for Long QT Syndrome, we now have an opportunity to address both the pro-arrhythmic and metabolic pathways of heart failure and atrial fibrillation in patients with underlying metabolic disease. We look forward to beginning our proof-of-concept studies next year.”
THRV-1268 is a novel and potent inhibitor of the Serum Glucocorticoid Kinase 1 (SGK1). SGK1 is a PI3-kinase-dependent kinase with its expression and activation regulated by several metabolic signaling pathways that are crucial in cardiometabolic diseases. Thryv Therapeutic’s first SGK1 inhibitor, LQT-1213, repeatedly demonstrated reductions in QTc interval in pre-clinical studies and in patients with the genetic and acquired Long QT Syndrome. SGK1 has been implicated in various pathological conditions, including QTc prolongation, arrhythmias, fibrosis, and heart failure. SGK1 dysfunction is also linked to cardiometabolic stressors such as obesity and hypertension, which are common comorbidities in heart failure.
Genetic and pharmacological inhibition of SGK1 has been demonstrated to have a protective effect in a mouse model of obesity-related atrial fibrillation. In several preclinical models of heart failure, THRV-1268 mitigated the development of heart failure and fibrosis, and reduced heart failure biomarkers associated with adverse changes in hemodynamic and functional cardiovascular outputs. These findings demonstrate the potential of SGK1 inhibition in addressing the pathogenesis of heart failure and related cardiometabolic conditions, including atrial fibrillation. Results from the THRV-1268 non-clinical efficacy program are to be presented at the American Heart Association meeting on November 16 – 18, 2024 in Chicago, Illinois.
Heart Failure
Heart failure is a complex clinical syndrome characterized by the heart’s inability to pump sufficient blood to meet the body’s needs. It can manifest in two primary forms: Heart Failure with Reduced Ejection Fraction (HFrEF), where the heart muscle is weakened and cannot contract effectively, leading to a reduced ejection fraction; and Heart Failure with Preserved Ejection Fraction (HFpEF), where the heart muscle contracts normally but is stiff and unable to relax properly, resulting in impaired filling and increased pressure in the heart. Often coexisting with atrial fibrillation, heart failure can result from various underlying conditions such as coronary artery disease, hypertension and previous heart attacks. If left untreated, heart failure leads to symptoms like fatigue and shortness of breath that profoundly impact a patient’s quality of life, and significantly increases the risk of severe complications and mortality.
Atrial Fibrillation
Atrial fibrillation (AF or AFib) is a prevalent cardiac arrhythmia characterized by irregular and often rapid heartbeats. Affecting nearly 40 million individuals worldwide, AF significantly diminishes quality of life and heightens the risk of stroke, heart failure and other cardiovascular complications. Obesity and metabolic disorders are key risk factors for AF, contributing to its rising prevalence. Effective treatment strategies aim to control heart rate, restore normal heart rhythm and mitigate complications.
About Thryv Therapeutics Inc.
Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop potent and highly selective inhibitors of serum glucocorticoid inducible kinase 1 (SGK1) to treat Long QT Syndrome, heart failure, and atrial fibrillation. For more information, please visit www.thryvtrx.com.
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced positive 120-day data from its Phase 2a study evaluating CNP-104 as a potential treatment for patients with Primary Biliary Cholangitis (PBC), a chronic liver disease that is estimated to affect approximately 130,000 individuals in the U.S.
“These data highlight the potential of CNP-104 to be the first disease-modifying treatment for people living with PBC,” said Paul M. Peloso, M.D., Chief Medical Officer of COUR Pharmaceuticals. “In addition to safety and tolerability data supporting further studies, we observed multiple immunological and clinical measurements supporting mechanistic proof of concept for CNP-104. Additionally, we observed positive clinical endpoints such as a reduction in liver stiffness measured on Fibroscan in the active arms compared to placebo, with Fibroscan being a surrogate for reductions in fibrosis. These data suggest that CNP-104 has the potential to halt disease progression which would be a transformational advancement for people living with PBC. We look forward to presenting these encouraging data at an upcoming scientific meeting.”
Data Highlights:
CNP-104 led to a slowing of disease progression in liver stiffness on Fibroscan, reaching a statistically significant decrease on day 120 in the active arms compared to placebo (p = 0.011)
Study subjects in the placebo arm experienced a greater trending decrease in albumin levels compared with CNP-104 treated patients
CNP-104 demonstrated a favorable T cell response in pathogenic CD4 T cell populations and tolerance inducing CD8 T cells
Notably, Th17 T cells decreased in both number and percentage mean change, with statistically significant higher rates of response in the active arms versus placebo at day-120 (p = 0.0037)
CNP-104 was safe and well tolerated, and all drug related adverse events (AEs) were mild (Grade 1 or 2) and no drug related severe adverse events (SAEs) were reported
The Phase 2a first-in-human, proof-of-concept, randomized clinical trial (NCT05104853) is designed to assess the safety, tolerability, pharmacodynamics, and efficacy of CNP-104 in patients aged 18-75, unresponsive to treatment with ursodeoxycholic acid (UDCA) and/or obeticholic acid (OCA). Participants received 4 mg/kg or 8 mg/kg of CNP-104 or placebo on Days 1 and 8 with all patients receiving two I.V. loading doses administered one week apart. Of the 41 subjects dosed, the final randomization ratio approximated 1:1:1 placebo to 4 mg/kg CNP-104 to 8 mg/kg CNP-104. This study report considers the 120-day primary study period while the 20-month long-term safety evaluation is ongoing.
Christopher Bowlus, M.D., the Lena Valente Professor and Chief, Division of Gastroenterology and Hepatology at UC Davis, and Principal Investigator for the study, added, “Despite the recent evolution of the PBC treatment landscape, there remains a significant unmet need for new treatments. Unlike CNP-104, current therapies do not address the root cause of the disease. While these data are early and from a small number of patients, they are incredibly promising and merit future investigation.”
Detailed results from the Phase 2a study of CNP-104 will be submitted for presentation at a future scientific conference.
About CNP-104:
CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic activated PDC-E2 T-cells that drive inflammation in bile ducts, leading to improvement in clinical outcomes of liver health. In January 2022, CNP-104 received Fast Track Designation from the United States Food and Drug Administration (FDA), making it potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met.
About PBC:
Primary biliary cholangitis (PBC) is a chronic, life-threatening autoimmune disease of the liver that primarily affects women between 40 and 60 years old. PBC is one of the leading causes of liver transplant in women. People with PBC experience impaired bile flow (cholestasis) and accumulation of toxic bile acids in the liver, which can progress to fibrosis, cirrhosis and liver failure. Increasing fibrosis results in cirrhosis and eventual liver failure and the need for a transplant. Additional symptoms of PBC include fatigue and pruritus, which can have a significant impact on the quality of life for patients. While the exact cause of PBC is unknown, the presence of antimitochondrial antibodies (AMA) which target the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), are highly associated with the development of the disease.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.
— In preclinical models, small molecule DPX-101 demonstrated highly selective target activation and expected electrophysiological effects as well as improved cognitive performance in behavioral tests of frontal cortex and hippocampal functions —
TORONTO, November 4, 2024— Damona Pharmaceuticals, a clinical-stage biopharmaceutical company focused on discovering and developing small molecules for the treatment and prevention of cognitive deficits associated with brain disorders, announced today that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for DPX-101, a positive allosteric modulator (PAM) that selectively augments the function of the α5-GABA-A receptor (α5-GABAAR) in the brain for the potential treatment of cognitive deficits in Major Depressive Disorder (MDD) and other brain disorders. The company has planned a Phase 1 clinical trial to evaluate safety, tolerability, and pharmacokinetic and pharmacodynamic characteristics of DPX-101 in healthy adults.
“The FDA clearance of the DPX-101 IND allows us to initiate the Phase 1 clinical study of our lead asset, which promises to establish a new standard of care for the treatment of cognitive deficits associated with brain disorders,” said John Reilly, CEO of Damona. “Based on data from our preclinical studies and the compound’s selectivity for α5-GABAAR, we believe that DPX-101 could benefit a broad range of patients suffering from diseases that cause cognitive impairment. We look forward to the planned initiation of our Phase 1 clinical trial later this year.”
The Phase 1 randomized, double-blind, placebo- and comparator-controlled, single- and multiple-ascending dose (SAD/MAD) clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single- and multiple-ascending doses of DPX-101 in healthy adult participants. Part 1, a single-ascending dose (SAD) study, will enroll participants randomized into one of five planned cohorts. A parallel, open-label, comparator cohort will be treated with the α1-GABA-A receptor agonist Zolpidem to provide additional evidence on DPX-101 α5-GABAAR selectivity. Part 2, a multiple-ascending dose (MAD) study, will involve participants randomized into three escalating dose cohorts, each to receive daily administrations of DPX-101 or placebo for seven days. The planned primary endpoints are safety and tolerability of single and multiple oral doses of DPX-101. Planned secondary endpoints will assess the PK profile of DPX-101. Exploratory endpoints include the effect of DPX-101 on functional target engagement and key differentiation measurements compared to the effects of Zolpidem. The planned Phase 1 trial will use quantitative electroencephalogram (qEEG) as a biomarker to assess target engagement.
About DPX-101
DPX-101 is an investigational positive allosteric modulator (PAM) that selectively targets the α5 subunit of the GABA-A receptor (α5-GABAAR) to enhance its function. The α5-GABAAR subtype is a promising target for reversing cognitive impairment due to its location in the hippocampus and frontal cortex regions of the brain and its unique role in somatostatin-containing GABAergic interneuron tonic inhibition within microcircuits critical for healthy cognition. In preclinical studies, DPX-101 demonstrated highly selective activation of the α5-GABAAR and a broad therapeutic window with no side effects. In preclinical models of stress, depression, aging, and Alzheimer’s disease, DPX-101 reversed memory deficits and restored connections between brain cells. In a preclinical model of schizophrenia, DPX-101 reversed hyperactivity of dopamine neurons, a hallmark of schizophrenia. DPX-101 is orally available, passes the blood-brain barrier, and achieves appropriate plasma and brain levels.
About Damona Pharmaceuticals
Damona is a clinical-stage biopharmaceutical company dedicated to discovering and developing transformational precision medicines for patients suffering from cognitive deficits associated with brain disorders based on deep neuroscience knowledge of cortical microcircuits in health and disease. The company’s founder and collaborators have made pioneering discoveries regarding the unique role of the α5-GABA-A receptor subtype in cognition and its potential for reversing cognitive deficits in models of various brain disorders. Damona’s lead clinical program, DPX-101, is designed to reverse cognitive impairment and restore connections between brain cells. Learn more at www.damonapharma.com.
Contact:
Mary Moynihan M2Friend Biocommunications +1 (802) 951-9600 mary@m2friend.com
Edesa Biotech, Inc. (Nasdaq:EDSA), a clinical-stage biopharmaceutical company focused on developing host-directed therapeutics for immuno-inflammatory diseases, today announced that the company has entered into a purchase agreement with an entity affiliated with Par Nijhawan, MD, Edesa’s Chief Executive Officer and Founder, to invest up to $5.0 million in the company, including an immediate investment of approximately $1.5 million.
The entity will purchase shares of the company’s Series A-1 Convertible Preferred Shares, as amended (the “Series A-1 Preferred Shares”), having a stated value of $10,000 per share, and warrants (the “Warrants”) to purchase the company’s common shares in a transaction structured as an at-the-market issuance under Nasdaq rules. The Series A-1 Preferred Shares will be convertible into the company’s common shares at a conversion price of $3.445. The Warrants will be exercisable for a number of common shares equal to 75% of the common shares initially issuable upon the conversion of the Series A-1 Preferred Shares.
Commenting on today’s announcement, Dr. Nijhawan said “I am pleased to demonstrate my strong belief in Edesa’s future growth opportunities and my continuing commitment to lead the company’s strategic initiatives. I believe Edesa has a strong development pipeline, and I am confident that we can continue to build on our operational and clinical success.”
Subject to certain exceptions and adjustments for share splits, each Series A-1 Preferred Share is convertible into a number of Edesa’s common shares calculated by dividing (i) the sum of the stated value of the Series A-1 Shares being converted plus a return equal to 10% of such stated value per annum by (ii) the conversion price. The Warrants will have an exercise price of $3.445 per share, will be exercisable immediately upon issuance and will expire five years from the date of issuance. Under applicable Canadian securities laws, purchases of Series A-1 Preferred Shares under the purchase agreement in an aggregate amount greater than $2.0 million will be subject to the prior approval of the company’s shareholders, excluding Dr. Nijhawan. The company also announced that in connection with the transactions contemplated by the purchase agreement, the $10 million revolving credit agreement previously entered into with the purchaser has been terminated. The company did not draw any funds from the facility.
The securities described above have not been and will not be registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.
About Edesa Biotech, Inc.
Edesa Biotech, Inc. (Nasdaq: EDSA) is a clinical-stage biopharmaceutical company developing innovative ways to treat inflammatory and immune-related diseases. Its clinical pipeline is focused on two therapeutic areas: Medical Dermatology and Respiratory. In Medical Dermatology, Edesa is developing EB06, an anti-CXCL10 monoclonal antibody candidate, as therapy for vitiligo, a common autoimmune disorder that causes skin to lose its color in patches. Its medical dermatology assets also include EB01 (1.0% daniluromer cream), a Phase 3-ready asset developed for use as a potential therapy for moderate-to-severe chronic Allergic Contact Dermatitis (ACD), a common occupational skin condition. The company’s most advanced Respiratory drug candidate is EB05 (paridiprubart), which is being evaluated in a U.S. government-funded platform study as a treatment for Acute Respiratory Distress Syndrome (ARDS), a life-threatening form of respiratory failure. The EB05 program has been the recipient of two funding awards from the Government of Canada to support the further development of this asset. In addition to EB05, Edesa is preparing an investigational new drug application (IND) in the United States for EB07 (paridiprubart) to conduct a future Phase 2 study in patients with pulmonary fibrosis. Sign up for news alerts. Connect with us on X and LinkedIn.
Edesa Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by the use of words such as “anticipate,” “believe,” “plan,” “estimate,” “expect,” “intend,” “may,” “will,” “would,” “could,” “should,” “might,” “potential,” or “continue” and variations or similar expressions, including statements related to: the company’s belief that Dr. Nijhawan’s investment is strategic and demonstrates a strong belief in Edesa’s future growth opportunities and a continuing commitment to lead the company’s strategic initiatives; the company’s belief that it has a strong development pipeline; the company’s confident belief that it can continue to build on its operational and clinical success; and the company’s timing and plans regarding its clinical studies in general. Readers should not unduly rely on these forward-looking statements, which are not a guarantee of future performance. There can be no assurance that forward-looking statements will prove to be accurate, as all such forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause actual results or future events to differ materially from the forward-looking statements. Such risks include: the ability of Edesa to obtain regulatory approval for or successfully commercialize any of its product candidates, the risk that access to sufficient capital to fund Edesa’s operations may not be available or may be available on terms that are not commercially favorable to Edesa, the risk that Edesa’s product candidates may not be effective against the diseases tested in its clinical trials, the risk that Edesa fails to comply with the terms of license agreements with third parties and as a result loses the right to use key intellectual property in its business, Edesa’s ability to protect its intellectual property, the timing and success of submission, acceptance and approval of regulatory filings, and the impacts of public health crises. Many of these factors that will determine actual results are beyond the company’s ability to control or predict. For a discussion of further risks and uncertainties related to Edesa’s business, please refer to Edesa’s public company reports filed with the U.S. Securities and Exchange Commission and the British Columbia Securities Commission. All forward-looking statements are made as of the date hereof and are subject to change. Except as required by law, Edesa assumes no obligation to update such statements.
Contact: Gary Koppenjan Edesa Biotech, Inc. (289) 800-9600
COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the first patient has been dosed in its Phase 1b/2a proof-of-concept clinical trial evaluating CNP-106 as a potential treatment for people living with generalized myasthenia gravis (gMG).
“Currently approved treatments for gMG work via broad immunosuppression and do not address the underlying autoimmunity of the disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-106 has demonstrated a unique ability to induce tolerance to the acetylcholine receptor (AChR), leading to improved muscle function in an experimental model of autoimmune myasthenia gravis. These results indicate that CNP-106 has the potential to be a first-in-class treatment that offers people living with gMG a disease modifying therapy without burdensome immune system suppression.”
The Phase 1b/2a double-blind, randomized, placebo-controlled clinical trial (NCT06106672) will enroll up to 54 patients with gMG, aged 18-75, to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106.
About CNP-106
CNP-106 is a biodegradable nanoparticle that encapsulates acetylcholine receptor (AChR). gMG is primarily driven by the production of antibodies against AChR in the bloodstream. These antibodies either block or destroy the receptors for acetylcholine, which is a crucial neurotransmitter for muscle contraction. As gMG progresses, it may lead to serious health issues, such as myasthenic crisis, tumors of the thymus gland, thyroid imbalances, and other autoimmune diseases. CNP-106 is designed to potentially induce the downregulation of AChR T cell populations and AChR antibodies, leading to improved muscle function.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human clinical studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.
Axonis Therapeutics, a biotechnology company focused on the development of novel neuromedicines, announced today the successful completion of an oversubscribed $115 million Series A financing. Proceeds from the financing will be used to advance Axonis’ lead development candidate, AXN-027, through clinical proof-of-concept in patients. AXN-027 is a first-in-class oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain. Proceeds will also enable the development of next generation compounds targeting KCC2 in these and other indications, including psychiatric and neurodevelopmental disorders.
Cormorant Asset Management and venBio Partners co-led the financing with significant investments from Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures and Solasta Ventures. In conjunction with this financing, Axonis named Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant Asset Management and Jonathan Leff, M.D., Executive Partner, Private Equity, from Sofinnova Investments to its Board of Directors. Eos Bioinnovation, Hatteras Venture Partners, SCI Ventures, Alexandria Venture Investments, Tachyon Ventures, BoxOne, Iaso Ventures and Pathway Bioventures also participated in the financing.
“Axonis’ vision is to advance a pipeline of oral KCC2 therapeutics to restore functional inhibition in the CNS to treat neurological disorders without disabling side effects,” said Joanna Stanicka, Ph.D., Chief Executive Officer, President, and co-founder of Axonis. “As we head into the clinic later this year, this financing is a significant milestone for Axonis as we translate our exciting science into efficacious and well-tolerated treatments for patients suffering from neurological disorders.”
“We’re delighted that this accomplished group of investors share our mission to translate Axonis’ science into impactful medicines,” said Corey Goodman, Ph.D., Executive Chair and co-founder of Axonis. “I’m pleased to see how far the science has progressed at Axonis; what the team has accomplished is remarkable. We are now well positioned to bring our lead therapeutic program through clinical proof of concept and expand into related arenas and build a leading biotechnology company.”
Despite the approval of several drugs for epilepsy and pain, many patients do not tolerate or respond to current first-line therapies due to impaired synaptic inhibition, which can contribute to drug resistance. Axonis’ oral KCC2 potentiators are designed to restore functional inhibition and overcome drug resistance in patients.
“I’m enthusiastic about the potential of Axonis’ novel precision approach to re-shape how we treat patients with neurological disorders, particularly epilepsy and pain where modulation of synaptic inhibition has great therapeutic promise,” said Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant. “Axonis’ scientists have done amazing work. We’re very excited about what this program could mean for patients and their families.”
Leadership Team
Axonis is led by Joanna Stanicka, Ph.D., co-founder and co-inventor of Axonis’ underlying IP. She is a Harvard-trained scientist, drug hunter, and entrepreneur, and a recipient of the Massachusetts Next Generation Award, a prize in leadership for woman-CEO in life sciences. Shane Hegarty, Ph.D., is the company’s Chief Scientific Officer, co-founder, and IP co-inventor. He was previously a Research Fellow at Harvard Medical School and Boston Children’s Hospital, faculty member in Neuroscience Department of University College Cork, and recipient of the Neuroscience Ireland Investigator Award.
In conjunction with the financing, Axonis also announces the appointments of Donald Manning, MD, Ph.D., as Chief Medical Officer and Jeff Imbaro, M.B.A., as Chief Operating Officer. Dr. Manning is a clinician-scientist with comprehensive experience in acute and chronic clinical practice, research and management. He brings to Axonis over 25 years of executive experience, having overseen the filings of seven INDs and five NDAs that have led to nine approved products. Mr. Imbaro brings over 20 years of company building expertise, having led Business Development and General and Administrative functions of several successful biotech and pharmaceutical companies. He was a member of the founding team of Semma Therapeutics, which was later acquired by Vertex Pharmaceuticals in 2019 for nearly $1 billion.
About Axonis Therapeutics
Axonis Therapeutics is a neuro-focused biotechnology company developing first- and best-in-class medicines targeting KCC2, the critical mediator of inhibitory neurotransmission within the brain, by translating breakthrough discoveries spun out from Boston Children’s Hospital, Harvard and Université Laval. The company has built a proprietary KCC2 discovery engine, based on several years of world-leading know-how, to become the leaders in this potential blockbuster drug space. Axonis is grateful for grant awards and support received from National Institutes of Health, Department of Defense SCIRP, ISS National Lab, CURE Epilepsy, Wings for Life, Massachusetts Life Center, SynGAP Research Fund, Praxis Spinal Cord Institute, Creative Destruction Lab, Springboard Enterprises and MassChallenge. The company is headquartered in Boston, MA. For more information, visit www.axonis.us.
About venBio Partners
Since inception in 2011, venBio has raised roughly $2 billion in capital commitments and led investment rounds in 40+ companies, including Labrys Biologics (acquired by Teva), Aragon Pharmaceuticals (acquired by Johnson & Johnson), Seragon Pharmaceuticals (acquired by Roche), Aurinia Pharmaceuticals (NASDAQ: AUPH), Apellis Pharmaceuticals (NASDAQ: APLS), Checkmate Pharmaceuticals (NASDAQ: CMPI, acquired by Regeneron), Turning Point Therapeutics (NASDAQ: TPTX, acquired by BMS), Akero Therapeutics (NASDAQ: AKRO), Harmony Biosciences (NASDAQ: HRMY), Pharvaris (NASDAQ: PHVS), CinCor Pharma (NASDAQ: CINC, acquired by Astra Zeneca), RayzeBio (NASDAQ: RYZB, acquired by BMS), and many others. For more information, visit the venBio website at www.venbio.com and follow on LinkedIn.
About Cormorant Asset Management
Founded in 2013, Cormorant Asset Management, LP (“Cormorant”) is a leading life sciences-focused investment firm with over $3 billion of assets under management. Cormorant is driven by a deep focus on fundamental scientific principles and provides financial resources to support the most innovative and promising publicly traded and private companies in biotech and allied sectors. Cormorant is among the most active crossover investors in life sciences, with investments in over 100 privately held, life science-focused companies during the past decade.
HistoSonics, (www.histosonics.com), the developer and manufacturer of the Edison Histotripsy System, announced today they have been awarded an exclusive contract to provide Veteran’s Affairs hospitals across the United States access to their novel non-invasive tumor liquefying platforms. The contract, the work of the Veterans Health Administration (VHA) and the Strategic Acquisition Center (SAC), establishes a $90 million ongoing schedule to develop histotripsy programs at key VA Hospitals. The announced VA contract is the culmination of a multi-year effort by HistoSonics, the company called Project Hero, to ensure meaningful value for Veterans and their families, VHA clinicians, administrators, and caregivers.
Histotripsy is a novel form of focused ultrasound that uses high amplitude, very short pulses to create a “bubble cloud” that is designed to mechanically destroy and liquefy targeted liver tumors. These bubble clouds form and collapse in microseconds, creating mechanical forces strong enough to destroy tissue at cellular and sub-cellular levels in a non-invasive and non-thermal method. Histotripsy is a single-setting, outpatient procedure for most patients and offers a promising treatment option for patients with liver tumors, avoiding complications like infection and toxicity which are common for invasive surgical and radiation procedures. The company believes that the novel mechanism of action of their proprietary technology may provide significant advantages to patients, including the ability of the treatment site to recover and resorb quickly.
“We are pleased that Veterans suffering with tumors will soon have access to this innovative, non-invasive treatment at select VA facilities with interdisciplinary expertise,” said Patrick Malloy MD, Executive Director of the VHA National Radiology Program. Mark Wilson, MD, VHA National Director of Surgery, added, “Histotripsy is an innovative tool that will further enable VA specialists to continue to provide a full continuum of care to Veterans.”
Receiving FDA “De Novo” clearance in October 2023, the Edison Histotripsy System has been successfully put into clinical practice at leading US academic centers and integrated health systems, as well as many community hospitals seeking to offer breakthrough histotripsy treatment for their patients. Michael Kelly MD, Executive Director of the VHA National Oncology Program, acknowledged the promise histotripsy has within the VA system and in conjunction with ongoing Federal oncology initiatives, “As the largest integrated oncology provider in the United States, Veterans Affairs remains committed to improving the lives of Veterans and supports the goals of collaborative programs like the Cancer Moonshot Initiative (https://www.cancer.va.gov/cancer-moonshot.html),”.
“Too frequently, Veterans do not have early access to meaningful innovation in health care, so it has been a mission for our team to partner with the VA in bringing histotripsy to our Veterans as soon as possible,” said Mike Blue HistoSonics CEO and President. Blue adds, “Histotripsy’s unique mechanism of action and non-invasive approach have demonstrated a patient friendly procedure for destroying liver tumors allowing many patients to return home and, ideally, to their lives quickly after the procedure. We believe that histotripsy is especially well-suited as a therapy for those who served with honor and are due the best healthcare we can provide.” All participating VA hospitals will be eligible to enroll patients in HistoSonics’ prospective study and post market clinical program, called BOOMBOX, which aims to collect data across a broad number of clinical use cases, and liver tumor pathologies, observing the use of histotripsy across all stages of liver disease.
HistoSonics is proud to partner with FIDELIS Sustainability as the SDVOSB partner and exclusive distributor of histotripsy systems to the federal government. “This is an absolute medical technology landmark for US patient care, and we are honored to be a part of bringing the Edison System to the Veteran Hospitals & DoD medical centers around the world,” said Dustin Lee Fidelis President & CEO.
The Edison System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any disease including, but not limited to, cancer or evaluated any specific cancer outcomes (such as local tumor progression, 5-year survival or overall survival). The System should only be used by physicians who have completed training performed by HistoSonics, and its use guided by the clinical judgment of an appropriately trained physician. Refer to the device Instructions for Use for a complete list of warnings, precautions, and a summary of clinical trial results, including reported adverse events.
About HistoSonics
HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison Histotripsy System in the US and the HistoSonics Histotripsy System in select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.
Cancer Research UK, one of the world’s largest funders of cancer research, and KisoJi Biotechnology Inc., a company focussed on the discovery and development of antibody therapeutics, have signed a landmark agreement to bring KisoJi’s lead asset, KJ-103, into a first-in-human clinical trial. KJ-103 is a naked anti-TROP2 antibody that has been created by KisoJi using its proprietary antibody technology.
Under the agreement, Cancer Research UK’s Centre for Drug Development (CDD) will sponsor, design and deliver a Phase 1/2a clinical trial of KJ-103, in selected TROP2 expressing solid tumours. KisoJi will supply the antibody for the clinical trial and work with CDD to complete the preclinical package. Cancer Research Horizons, Cancer Research UK’s innovation arm, will manage the commercial relationship.
Unlike antibody drug conjugates (ADCs), KJ-103 does not require a cytotoxic payload but instead functions by recruiting immune cells to kill tumour cells. KJ-103 binds to TROP2 in a location distinct from where current TROP2 ADCs bind. It uses TROP2 as a way of directing macrophage activation and phagocytosis of the tumour cells expressing it, leading to tumour cell death.
KJ-103 may provide an alternative treatment option for TROP2-expressing cancers in which TROP2 ADCs have proven ineffective or are not suitable due to their toxicity profile. Tumour types expressing TROP2 include: colorectal, head and neck, ovarian, breast, bladder and lung cancers.
Lars Erwig, Director of the CDD, said: “We are excited to collaborate with KisoJi to bring KJ-103 into clinical development. This partnership aligns with our mission to explore innovative therapeutic approaches for hard-to-treat cancers. With KJ-103’s unique mechanism of action, which harnesses the body’s immune system without the potential toxicity of a drug conjugate, we hope to offer new treatment options for patients with TROP2-expressing solid tumours.”
David Young, co-founder and CEO of KisoJi said: “We are thrilled to be advancing KJ-103 into the clinic in partnership with Cancer Research UK. As the first naked antibody to target TROP2 cancers, KJ-103 is the first of a new wave of antibodies to come from our modernised technology platform that leverages AI grounded in biology to create transformative antibody therapeutics.”
About Cancer Research UK’s Centre for Drug Development Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 30 years, taking over 160 potential new anti-cancer agents into clinical trials in patients. Six of these new agents have made it to market, including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase 3 trials. Thirteen agents remain in active development with the potential to reach the market. It currently has a portfolio of 16 projects in preclinical development, Phase 1 or early Phase 2 clinical trials.
About KisoJi Biotechnology KisoJi is a Canadian biotechnology company that deploys the latest scientific and AI tools to conduct therapeutic antibody discovery in a fundamentally new way. KisoJi has developed a multi-species transgenic mouse to generate highly diverse single domain antibodies, as well as a modular multi-specific antibody scaffold with high stability and productivity. Most recently, KisoJi has used advanced AI tools to visualise the universe of all antibodies against a target in order to uncover novel biology and new therapeutic antibody capabilities for its partners and its own pipeline.
About KJ-103 KJ-103 is a single domain antibody that binds to a unique epitope on TROP2. It acts via effector cell mechanisms, including the activation of macrophages, to kill tumour cells. KJ-103 has been shown to have significant anti-tumour potency with no evidence of toxicity or resistance across a number of preclinical solid tumor models. KJ-103 was humanised by LifeArc.
Cancer Research UK Contact: Tim Bodicoat tim.bodicoat@cancer.org.uk +44 203 469 8300 +44 203 469 8301 (out of hours)
KisoJi Biotechnology Contact: Investors and Media Argot Partners Sam Martin/Cameron Willis KisoJi@argotpartners.com +1 212 600 1902
SOURCE KisoJi Biotechnology Inc.; Cancer Research UK
Alpha-9 Oncology Inc. (Alpha-9 or the Company), a clinical stage company developing radiopharmaceuticals to meaningfully improve the treatment of people living with cancer, today announced an oversubscribed $175 million Series C financing to support the progression of its pipeline. The financing was led by Lightspeed Venture Partners and Ascenta Capital. A selected syndicate of new investors – General Catalyst, a16z Bio + Health, RA Capital Management, Janus Henderson Investors, Delos Capital, Digitalis Ventures, Lumira Ventures and a healthcare fund managed by abrdn Inc. – joined the round, in addition to existing investors Frazier Life Sciences, Longitude Capital, Nextech Invest, BVF Partners LP, and Samsara BioCapital. Shelley Chu, head of Lightspeed Venture Partners’ healthcare team and Evan Rachlin, co-founder and managing partner of Ascenta Capital will join the Company’s Board of Directors.
Alpha-9 has built a diversified portfolio of clinical and discovery assets across both validated and novel targets. The Series C will fund human studies for the clinical stage assets and advancement of discovery stage assets to clinic-ready development candidates. Furthermore, the Series C will fund expanded R&D capabilities and continued investment in CMC and supply chain.
“Over the last few years, Alpha-9 has built a leading radiopharmaceutical company with a deep pipeline and robust infrastructure,” said Alpha-9 CEO, David Hirsch, MD, PhD. “The Series C is an exciting, significant milestone for us and will greatly accelerate our growth. We are thrilled to have the backing of a top-tier investor syndicate who share our belief in the potential of radiopharmaceuticals.”
“Alpha-9 is developing a differentiated portfolio that includes multiple radiopharmaceuticals with first in-class and best-in-class potential,” said Shelley Chu, head of Lightspeed Venture Partners’ healthcare team. “We are impressed with the team’s progress to date and are proud to support the advancement of these programs.”
Alpha-9’s approach to designing bespoke molecules is systematic and data-driven. The Company has a differentiated toolbox of binders, linkers, chelators and radioisotopes – elements that each play an integral role in radiopharmaceutical development. Alpha-9 designs each component of the radiopharmaceutical for optimal selectivity, stability and payload delivery. The Alpha-9 approach is rigorous, fast and capital efficient, generating best-in-class compounds for rapid clinical development.
“We have been following this space for a long time. What differentiated Alpha-9 was its effective approach to molecule design as well as its thoughtful strategy on infrastructure expansion,” said Evan Rachlin, MD, managing partner of Ascenta Capital. “We are pleased to support the Company’s continued progress as it strives to deliver on the promise of radiopharmaceuticals.”
To support its endeavors, Alpha-9 has purpose-built research facilities in Vancouver, which were completed last year and have been operating at scale. These facilities help to accelerate drug development by streamlining discovery processes. Alpha-9 has also partnered with isotope suppliers and CDMOs to support its ongoing clinical trials. Alpha-9’s commitment to continue building robust infrastructure and world class capabilities underscores the company’s mission to provide effective treatments for patients worldwide.
About Alpha-9 Oncology Inc.
Alpha-9 Oncology Inc. is a clinical stage radiopharmaceutical company developing differentiated and highly targeted radiopharmaceuticals with the potential to meaningfully improve the treatment of people living with cancer. Applying proprietary technologies and deep foundational expertise, Alpha-9 is on the forefront of engineering bespoke radiopharmaceuticals that are optimized to selectively deliver radiation to tumor sites while minimizing off-target effects. Alpha-9 is advancing a robust pipeline of novel radiopharmaceuticals with a systematic approach to molecule design that offers broad potential for expansion into several validated oncology targets. For more information, please visit www.a9oncology.com.
About Lightspeed Venture Partners
Lightspeed Venture Partners is a multi-stage venture capital firm focused on accelerating disruptive innovations and trends in the Enterprise, Healthcare, Consumer and Fintech sectors. Over the past two decades, the Lightspeed team has backed hundreds of entrepreneurs and helped build more than 500 companies globally including biotechnology companies such as Abata, Ancora, Diagonal, Enlaza, Forty Seven, Personalis, Pheon, Teneobio, Triana, Scorpion, Seismic, Ultima Genomics, Xaira, ZagBio, and more. Lightspeed and its global team currently manage $25B in AUM across the Lightspeed platform, with investment professionals and advisors in the U.S., Europe, India, Israel, and Southeast Asia. For more information, please visit www.lsvp.com.
About Ascenta Capital
Ascenta Capital is a biotech venture fund, co-founded in 2023 by Dr. Evan Rachlin and Dr. Lorence Kim. The fund is focused on a concentrated portfolio of investments in multi-product companies. For more information about Ascenta Capital, visit www.ascentacap.com.
Notice Regarding Forward-Looking Statements:
This news release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and forward-looking information within the meaning of applicable Canadian securities laws (collectively, “forward-looking statements”). The words “may”, “will”, “potential”, “believes” and “if” are intended to identify forward-looking statements, although not all forward-looking statements contain such terms. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause actual results, future circumstances, or events to differ materially from those projected in the forward-looking statements. These risks include but are not limited to: those associated with the success of research and development programs, the ability to raise additional funding, and the need to obtain regulatory approval. Forward-looking statements are made as of the date hereof and Alpha-9 disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for autoimmune diseases, today announced it will present detailed safety and efficacy results from the Phase 2a study of CNP-104 in Primary Biliary Cholangitis (PBC) via a late-breaking poster at The Liver Meeting® 2024, being hosted by the American Association for the Study of Liver Disease (AASLD) from November 15-18 in San Diego, California.
Presentation Details:
Title: Tolerogenic treatment with CNP-104 results in regulation Th17 cells slowing progression of PBC on liver stiffness
Presenter: Paul M. Peloso, M.D., COUR Chief Medical Officer
Publication Number: 5039
Poster Session Date and Time: Monday, November 18, 2024, from 1:00 to 2:00 p.m. PT
About CNP-104:
CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic PDC-E2 T-cells that drive inflammation in bile ducts, resulting in clinically improved outcomes in liver health. In January 2022, CNP-104 received Fast Track Designation from the United States Food and Drug Administration (FDA), making it potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met.
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.
COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.
enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), a clinical-stage genetic medicines company whose non-viral lead investigational product detalimogene voraplasmid (also known as detalimogene, and previously EG-70) is in an ongoing pivotal study in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis), today announced the appointment of Joan Connolly as Chief Technology Officer (CTO) and member of the corporate leadership team. In addition, enGene Co-Founder and former CTO, Anthony Cheung, Ph.D., will transition to the role of Chief Scientific Officer, succeeding James Sullivan, MSc, Ph.D.
I am excited to join enGene at this critical juncture. Detalimogene’s highly differentiated, non-viral DDX formulation and easily scalable manufacturing process are designed to offer significant advantages over other therapies available and in development.
Ms. Connolly’s career spans more than 30 years, with experience across CMC development for large and small molecules, manufacturing management, regulatory filings, and product commercialization, as well as supply chain, logistics and sourcing, and procurement. Ms. Connolly has extensive experience filing submissions for regulatory approval and product launches. In her most recent role, Ms. Connolly served as Chief Technology Officer of Albireo Pharma, Inc., where she oversaw drug substance and product development, clinical supply distribution, commercial supply chain and quality. Prior to Albireo, she held senior roles at Stemline Therapeutics, Inc., ImClone Systems Inc., and Bristol-Myers Squibb.
“We are thrilled to welcome Joan to the enGene team. She brings significant experience in managing the technical operations required to drive a late-stage clinical asset through to commercialization, which will be critical as we complete the pivotal portion of the LEGEND study of detalimogene and anticipate filing our BLA in mid-2026,” said Ron Cooper, Chief Executive Officer of enGene.
“I am excited to join enGene at this critical juncture. Detalimogene’s highly differentiated, non-viral DDX formulation and easily scalable manufacturing process are designed to offer significant advantages over other therapies available and in development,” said Ms. Connolly. “Recent preliminary data from LEGEND’s pivotal arm underscore detalimogene’s future potential as a foundational therapy for the many NMIBC patients in need of new therapeutic options.”
Dr. Cheung is a co-founder of enGene and has served in various capacities since the Company’s inception. He has been central to the discovery and development of the Company’s novel and proprietary Dually Derivatized Oligochitosan (DDX) platform and in the creation of detalimogene. He has co-authored numerous book chapters, review articles and peer-reviewed journals, and is a named inventor on numerous patents in the areas of gene therapy and polymer chemistry.
“Having developed enGene’s DDX technology from the ground up, Anthony is intimately familiar with the science and well-suited to drive future expansion of our DDX platform,” said Ron Cooper. “We are grateful to Dr. Sullivan for his meaningful contributions in advancing detalimogene and wish him luck in his future endeavors.”
About enGene
enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is detalimogene voraplasmid, (also known as detalimogene, and previously EG-70) for patients with Non-Muscle Invasive Bladder Cancer (NMIBC) – a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a pivotal cohort studying detalimogene in Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (Cis). Detalimogene was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. For more information, visit enGene.com.
Forward-Looking Statements
Some of the statements contained in this press release may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s expectations, hopes, beliefs, intentions, goals, strategies, forecasts and projections. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: our expectations regarding regulatory submissions, our beliefs as to the potential benefits of detalimogene, and our expectations regarding the potential broad adoption of detalimogene.
Many factors, risks, uncertainties and assumptions could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, that preliminary clinical data may not accurately reflect the complete results of a particular study and remain subject to audit and verification, and final data may differ materially from preliminary data; the Company’s ability to recruit and retain qualified scientific and management personnel; establish clinical trial sites and enroll patients in its clinical trials; execute on the Company’s clinical development plans and ability to secure regulatory approval on anticipated timelines; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2023 and most recent Quarterly Report on Form 10-Q for the fiscal quarter ended July 31, 2024 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).
You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.
Endogenex, Inc., a clinical-stage medical device company focused on improving outcomes for patients with type 2 diabetes (T2D), today announced the enrollment of the first patient in the ReCET Clinical Study. This multicenter, prospective, randomized, double-blinded, sham-controlled trial is designed to assess the safety and effectiveness of the ReCET™ System in adult patients whose T2D is inadequately controlled with non-insulin, glucose-lowering medications.
“This milestone is a major step forward in our mission to transform T2D treatment by addressing the underlying causes impacting millions of patients living with T2D,” stated Stacey Pugh, CEO of Endogenex. “We are thrilled to have completed the first enrollment and look forward to continued collaboration with our ReCET Clinical Study sites in advancing study enrollment.”
The first patient was successfully enrolled at Orlando Health, Orlando, FL, under the leadership of Dr. Andre Teixeira, Medical Director of the Orlando Health Weight Loss and Bariatric Surgery Institute. “We are excited to be part of this landmark study,” stated Dr. Teixeira. “Clinical evidence continues to evolve, supporting the central role of the duodenum in regulating glucose metabolism. Therapeutic interventions targeting the upper gastrointestinal tract are demonstrating improved outcomes for patients with type 2 diabetes. The ability to reset pathologic signaling in the duodenum using the ReCET Technology, delivered through an endoscopic procedure, holds great promise for transforming care for T2D patients.”
“We are proud to have reached this important milestone in the ReCET pivotal study,” stated Dr Richard Pratley, Medical Director of the Advent Health Diabetes Institute in Orlando, FL, and Co-Principal Investigator for the ReCET Study. “The ReCET Study offers new hope for advancing treatment options and the potential to provide patients a better opportunity to manage their diabetes more effectively.”
The ReCET Study is a pivotal trial approved by the U.S. Food and Drug Administration (FDA) under the Investigational Device Exemption (IDE). The study aims to enroll up to 350 patients at approximately 40 sites in the United States and Australia.
About the ReCET™ Procedure ReCET is a novel, endoscopic outpatient procedure that targets the cellular pathology of the duodenum. This pathology may contribute to the development and progression of type 2 diabetes.
The ReCET System aims to initiate the body’s natural regenerative process by applying highly controlled, non-thermal pulsed electric fields to the mucosa and sub-mucosa duodenal tissue. This approach may help restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels.
The ReCET System has been evaluated in feasibility clinical studies, such as REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. These studies assessed the safety and efficacy of the treatment in adults with type 2 diabetes whose blood glucose levels were inadequately controlled despite using insulin and non-insulin medications. Preliminary outcomes from these studies have been presented at medical conferences globally.
About Endogenex Endogenex, founded in partnership with Mayo Clinic, aims to revolutionize treatment options for individuals with type 2 diabetes (T2D). The company’s innovations focus on resetting the body’s metabolic signaling system by harnessing its natural regenerative capabilities to improve metabolic function. Through the development of the ReCET System and the novel application of precise, controlled, non-thermal pulsed electric fields, Endogenex is establishing a new era in T2D therapy, helping patients regain control of their blood glucose levels and slow disease progression.
Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD) have been accepted for both oral and poster presentation at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting. The hybrid meeting will take place October 21-24, 2024, in Clearwater, Florida, and virtually.
“Given the effects of TPN-101 on key biomarkers of neurodegeneration and neuroinflammation, including NfL and IL-6, and clinical outcome measures of disease progression and respiratory function in patients with C9orf72-related ALS, we are very pleased these data were accepted for presentation at NEALS,” said Andrew Satlin, M.D., Chief Medical Officer at Transposon. “Based on these promising results, we are advancing TPN-101 into a Phase 3 registration study for the treatment of C9orf72-related ALS, while also continuing to develop TPN-101 for other neurodegenerative diseases.”
About the Phase 2 Study in C9orf72-related ALS/FTD The Phase 2 study in patients with C9orf72-related ALS/FTD was a multi-center, randomized, double-blind, placebo-controlled parallel-group, two-arm study with an open-label treatment period. Participants (n=42) were randomized 3:2 to receive daily doses of 400 mg of TPN-101 or placebo. The study included a six-week screening period, a 24-week double-blind treatment period, a 24-week open-label treatment period, and a follow-up visit four weeks post-treatment. Further information on the study can be accessed at ClinicalTrials.gov.
About TPN-101 TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 cDNA, triggering innate immune responses that contribute to neurodegenerative, neuroinflammatory, and aging-related disease pathology.
About ALS and FTD ALS is a neurodegenerative disease characterized by progressive muscle weakness, and loss of ability to speak, eat, move or breathe. FTD is a progressive frontal/temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating, or working. A C9orf72 hexanucleotide repeat mutation accounts for 10-15% of both disorders. With onset commonly in middle age or earlier, patients with ALS have a mean survival of two to three years. Patients with FTD have a mean survival of nine years.
About Transposon Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications.
Contact: Rick Orr Transposon Therapeutics, Inc. (858) 535-4821 rorr@transposonrx.com
Thryv Therapeutics Inc., a clinical-stage biotechnology company, received positive top-line results from the Wave I Part 2 proof-of-concept clinical study evaluating LQT-1213 for the treatment of congenital Long QT Syndrome (cLQTS) Types 2 and 3. LQT-1213, a potent and selective serum glucocorticoid inducible kinase 1 (SGK1) inhibitor, demonstrated clinically meaningful and statistically significant reductions in the QTcF interval (a measure of how long it takes the heart to “reset” between beats) after only two days of dosing in patients with cLQTS Types 2 and 3. LQT-1213 appeared to be generally well-tolerated in the study with no drug-related adverse events (AEs) reported.
Congenital LQTS is a rare genetic condition that affects the heart’s electrical system, leading to an increased risk of life-threatening arrhythmias in children and adults. While Wave I Part 1 evaluated the effect of LQT-1213 in a model of LQTS induced by the drug dofetilide, Wave 1 Part 2 included, for the first time, patients with genetically confirmed congenital LQTS Types 2 and 3, providing direct clinical insights into the acute efficacy of LQT-1213 in the targeted patient population. It is believed that reductions in QTcF will reduce arrhythmic risk in pediatric and adult patients with prolonged QTc intervals.
“We are excited about the positive top-line results from the Wave I Part 2 study in cLQTS Types 2 and 3 patients. Two days of treatment with LQT-1213 demonstrated rapid and clinically meaningful reductions in QTcF. As well, a clear dose level effect was seen with the highest dose of LQT-1213 reducing QTcF for all key endpoints of the study,” said Philip Sager, MD, FAHA, Chief Medical Officer of Thryv Therapeutics. “The totality of LQT-1213 treatment effects, which are on top of beta blocker therapy, has allowed us to engage regulatory agencies about our next steps towards a pivotal program in patients with congenital Long QT Syndrome.”
Study Highlights
Wave I is a multi-part, proof-of-concept clinical study designed to evaluate the safety and efficacy of LQT-1213 in reducing QT in patients with both acquired and congenital forms of Long QT Syndrome. Further details about the study can be found at clinicaltrials.gov (NCT05906732).
Full data from the Wave I Part 2 study in congenital LQTS Types 2 and 3 will be presented at an upcoming scientific conference in 2025. Part 3 of the Wave I study will enroll patients with cLQTS Type 1 who demonstrate continued prolongation of their QT interval despite standard of care.
About LQT-1213
LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Types 1, 2, and 3. By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in children and adults with LQTS, thereby decreasing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating both congenital and acquired LQTS. LQT-1213 has been granted Orphan Drug Designation by the US FDA for the treatment of congenital Long QT Syndrome.
About Long QT Syndrome
LQTS, is a disorder of the electrical system that results in the heartbeat taking longer than usual to “reset” between beats, measured on the ECG as the QTc interval. LQTS can be either congenital or acquired due to certain drugs. Congenital LQTS is a set of rare orphan diseases in which people are genetically predisposed to chronic prolongation of their QTc interval (commonly more than 480 milliseconds), leading to increased risk of torsades de pointes, a lethal cardiac arrhythmia that can cause sudden cardiac death. Acquired Long QT may develop from the administration of therapies which block electrical pathways in the heart, leading to a similar mechanistic prolongation of QTc and risk of sudden cardiac death.
About Thryv Therapeutics
Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of serum glucocorticoid inducible kinase. For more information, please visit www.thryvtrx.com.
Thryv Therapeutics Inc., a clinical stage biotech company dedicated to developing treatments for rare and life-threatening cardiovascular indications, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to LQT-1213 for the treatment of Long QT Syndrome (LQTS). LQT-1213 is a novel, first-in-class SGK1 inhibitor specifically designed to treat congenital LQTS. Thryv has developed a series of SGK1 inhibitors for the treatment of cardiometabolic stress associated with various arrhythmic diseases including LQTS, atrial fibrillation, and heart failure.
“We are pleased with the FDA’s Orphan Drug Designation for LQT-1213 based partly on emerging positive clinical data from our Wave 1 clinical study in patients with Long QT Syndrome” said Debra Odink, PhD, President and Chief Development Officer of Thryv Therapeutics. “People with Long QT Syndrome deserve a properly studied and FDA-approved therapy to help in their battle against this potentially lethal genetic disease. There is currently no FDA-approved therapy for people with Long QT Syndrome. This designation reinforces the potential of LQT-1213 to fulfill this unmet need and provides critical incentives for Thryv to accelerate our efforts to deploy prospectively designed, pivotal efficacy studies in people with congenital Long QT Syndrome. We remain focused on bringing innovative treatments to adults and children with rare diseases who have limited options.”
Following a request from the FDA, clinical data from the ongoing Wave I clinical study in people with congenital LQTS was provided to the Office of Orphan Products Designation. Shortly thereafter, the company received notification that its request for Orphan Drug Designation was approved.
The FDA Orphan Drug Designation is a special status granted to drugs and biologics targeting rare diseases, defined as those affecting fewer than 200,000 people in the United States. This designation offers numerous benefits to support the development of treatments for rare conditions, including tax credits for clinical trial costs, exemption from certain FDA fees, and up to seven years of market exclusivity following FDA approval.
About LQT-1213
LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Type 1, 2, and 3. By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in people with LQTS, thereby decreasing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating congenital LQTS.
Additional information about the on-going Wave I clinical study is available at www.clinicaltrials.gov using identifier: NCT05906732.
About Thryv Therapeutics Inc.
Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit www.thryvtrx.com.
SpectraWAVE, Inc., a medical imaging company focused on improving the treatment and outcomes for patients with coronary artery disease (CAD), today announced a $50M Series B funding round led by Johnson and Johnson Innovation – JJDC, Inc. joined by S3 Ventures, Lumira Ventures, SV Health Investors, Deerfield Management, NovaVenture, Heartwork Capital, and undisclosed parties. The funding round will advance commercial expansion and product additions to the Company’s 510k-cleared HyperVue™ Imaging System.
“Intravascular imaging guidance is backed by a wealth of randomized clinical trials (RCT) and recently collated in a meta-analysis of 22 RCT studies and approximately 16,000 patients published this year in The Lancet, demonstrating significantly improved outcomes for patients undergoing coronary stenting,” said Eman Namati, Ph.D., Chief Executive Officer of
SpectraWAVE. “This evidence and broad recognition of impact has recently moved intravascular imaging to a 1A guideline recommendation in Europe, with an expectation that the United States will follow in due time. Increasing both the capabilities and the ease of use of intravascular imaging systems is now critical to expand the use of imaging and improve care for these patients. That is our mission with HyperVue – simpler, faster, and better imaging to drive optimized stenting procedures for improved patient outcomes. The reception to the product during our initial U.S. launch has been fantastic, and this financing, supported by leading MedTech investors, will support our commercial expansion to bring HyperVue to more patients.”
Intravascular imaging is an essential tool to optimize coronary stenting procedures, providing key insights into plaque morphology, plaque modification decisions, stent and balloon sizing and landing zone selection, confirmation of treatment optimization, and future adverse event risk. Prior intravascular imaging technologies offer compromises in image resolution, image depth, and ease of use. HyperVue is the first intravascular imaging technology to combine two important imaging technologies, DeepOCT and NIRS, while optimizing for image quality and procedural efficiency in the cath lab, including no-flush catheter prep, fast and long pullbacks designed to reduce and remove the use of contrast, and the most comprehensive AI-driven workflow and image analysis offering.
Namati continued, “In addition to our commercial intravascular imaging system, we are developing a wire-free physiology software add-on to allow physicians rapid assessment of pressure drops in the coronaries using the same HyperVue hardware. This is an important step in our journey to enhance the clinical decision-making for these patients and establish an anchor point for future innovation in the cath lab.”
About SpectraWAVE, Inc.
SpectraWAVE, located in Bedford, Mass., is a privately held medical device company founded in 2017 to provide unrivaled optical and computational insights to improve the treatment and outcomes for patients with coronary artery disease (CAD). CAD, the buildup of plaque in the wall of the arteries that supply blood to the heart, affects 20.5 million adults aged 20 and older. In CAD patients that undergo percutaneous stent placement, it is estimated that one in five patients experience adverse events within two years. SpectraWAVE’s flagship HyperVueTM Imaging System combines next generation DeepOCTTM images and near infrared spectroscopy (NIRS) with workflows optimized for the cardiac catheterization lab, and serves as a central hub for future enhancements that will continue to empower interventionalists in their treatment decision making and optimization. The HyperVue Imaging System is intended for the imaging of coronary arteries and is indicated in patients who are candidates for transluminal interventional procedures. The NIRS capability of the system is intended for the identification of patients and plaques at increased risk of major adverse cardiac events. For more information and complete indications for use, please visit www.spectrawave.com.
HyperVue, Starlight, and DeepOCT are commercial trademarks cleared for sale in the U.S.A.
HistoSonics, (www.histosonics.com), the developer and manufacturer of the Edison™ Histotripsy System, announced today the company’s initial partnership in Asia with Hong Kong University receiving their first histotripsy system after a generous donation from the Li Ka Shing Foundation. The Foundation, led by its founder Mr. Li Ka-shing, is a prolific global philanthropic organization with a focused effort in Hong Kong, and is responsible to date for over hk$30 billion in projects involving education, medical services, charity, and anti-poverty programs. As part of the Foundation’s focus on bringing innovative medical advancements to Hong Kong, the Foundation provided the capital to acquire two initial histotripsy systems to the leading public hospitals in Hong Kong, one each to the University of Hong Kong and the Chinese University of Hong Kong. Chinese University of Hong Kong will receive the second histotripsy system early next year to complete the initial gift from Mr. Li.
Mr. Li Ka-shing Gifts Histotripsy Systems to Hong Kong Hospitals with Liver Team, 27 Aug. 2024
Histotripsy is a novel form of focused ultrasound that uses high amplitude, very short pulses to create a “bubble cloud” that is designed to mechanically destroy and liquefy targeted liver tumors. These bubble clouds form and collapse in microseconds, creating mechanical forces strong enough to destroy tissue at cellular and sub-cellular levels in a non-invasive and non-thermal method. Histotripsy offers a promising alternative for patients with liver tumors to avoid certain side effects like surgical site infections, bleeding, and radiation toxicity common to other treatments such as surgery and radiation therapy. The company believes that the novel mechanism of action of their proprietary technology may provide significant advantages to patients, including the ability of the treatment site to recover and resorb quickly. Mr. Li commented, “Treating tumors using the HistoSonics Histotripsy System by forming microbubbles is fascinating” and he believes medical research and innovation can bring hope and economic prospects to the city.
Experts in liver treatment from the University of Hong Kong and the Chinese University of Hong Kong recently underwent training at HistoSonics headquarters in Minneapolis, MN and expect to begin treating patients as early as this week. “We could not be more excited to partner with these two prestigious university hospitals to be the first in Hong Kong to offer our novel therapy to their patients,” said Mike Blue, HistoSonics CEO and President. “Unfortunately, Asia carries a substantial proportion of the world’s population suffering from diseases that cause liver tumors and based on the experiences from our U.S. launch we are confident that we can begin to make an immediate impact on the quality of life of many of the patients who need it most in this region. We are very grateful for the Foundation’s very generous gift and Mr. Li’s personal interest in helping patients in Hong Kong,” added Blue. Both Hong Kong based hospitals will be eligible to enroll patients in HistoSonics’ prospective study and post market clinical program, called BOOMBOX, which aims to collect data across a broad number of clinical use cases, and liver tumor pathologies, observing the use of histotripsy across all stages of liver disease.
The Edison Histotripsy System is indicated for the non-invasive destruction of liver tumors, including unresectable liver tumors, using a non-thermal, mechanical process of focused ultrasound.
About HistoSonics
HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison Histotripsy System in the US and the HistoSonics Histotripsy System in select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.
“The pooled #HOPE4LIVER single-arm pivotal trial for histotripsy of primary and metastatic liver tumors” published in Radiology by Mishal Mendiratta-Lala, et.al. is licensed under CC BY 4.0.
Contacts
For Media Inquiries Media contact: Josh King Vice President of Marketing Email : Joshua.king@histosonics.com Phone : 608.332.8124
HistoSonics, the manufacturer of the Edison® Histotripsy System and novel histotripsy therapy platforms, announced today the completion of an oversubscribed $102 million Series D financing. The round was led by Alpha Wave Ventures, a world leader in growth stage investments, with participation from new investors Amzak Health and HealthQuest Capital, and existing investors Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Venture Investors, Lumira Ventures, Yonjin Venture, the State of Wisconsin Investment Board, and others. Histotripsy is a novel form of focused ultrasound that uses high amplitude, very short pulses to create a “bubble cloud” that is designed to mechanically destroy and liquefy targeted liver tumors. These bubble clouds form and collapse in microseconds, creating mechanical forces strong enough to destroy tissue at cellular and sub-cellular levels in a non-invasive and non-thermal method. Histotripsy offers a promising alternative to treatments such as surgery, radiation and chemotherapy, which often have significant side effects. The Series D funding will be used to accelerate category defining advancements to its non-invasive histotripsy platforms, support commercial growth in the US and in planned global markets, and initiate the company’s innovative, prospective BOOMBOX Master Study that will evaluate HistoSonics’ Edison System for the treatment of liver tumors across multidisciplinary users. The Edison Histotripsy System was granted FDA De Novo clearance in October 2023. HistoSonics is currently partnering with leading institutions across the US and internationally in developing multi-disciplinary histotripsy liver programs. “Histotripsy is a paradigm-changing treatment option for patients who want a non-invasive approach to target and destroy tumors without the need for needles or incisions,” said Mike Blue, President & CEO, HistoSonics. “We’re thrilled to announce this top-tier investor syndicate led by Alpha Wave, which reinforces the confidence in our mission to impact patients’ lives with our current liver application and expanded use in kidney, pancreas, prostate, brain and other tumor types. This funding will accelerate key projects designed to enhance core technical capabilities impacting current and future platforms, and support collaboration with physicians and researchers studying innovative ways to use histotripsy’s unique mechanism of action to improve patient outcomes. By pioneering a new category of medicine with histotripsy, HistoSonics is redefining liver tumor treatment and providing hope for millions of people worldwide who desperately need new options.” “Alpha Wave is proud to lead the investment in HistoSonics, a pioneer in the use of histotripsy,” said Chris Dimitropoulos, Managing Director, Healthcare Investments at Alpha Wave Global. “Histotripsy’s unique non-invasive approach uses focused ultrasound to precisely target and destroy diseased tissue without damaging surrounding healthy tissue. This groundbreaking technology has the potential to transform the treatment landscape for a variety of challenging clinical conditions, offering patients improved outcomes and recovery times. The level of adoption we’ve seen from leading hospital centers across the country and overseas highlights the huge unmet medical need. We are excited to support HistoSonics in accelerating the development and availability of this innovative therapy.” In connection with this financing, Mr. Dimitropoulos will join the HistoSonics Board of Directors. HistoSonics’ Edison System uses proprietary technology and advanced imaging to deliver personalized, non-invasive histotripsy treatments with precision and control. The novel mechanism of action of this proprietary technology may provide significant patient advantages, including the ability of the treatment site to recover and resorb quickly. HistoSonics’ platform also provides physicians the ability to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today. About HistoSonics HistoSonics is a privately held medical device company developing non-invasive platforms and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, prostate, brain, and others. HistoSonics has offices in Ann Arbor, Michigan, and Minneapolis, Minnesota. The Edison® System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any specific disease or condition. Patients seeking information on histotripsy and if they may be a candidate for histotripsy can learn more at www.myhistotripsy.com. Use of the Edison System in kidney applications is limited by federal law to investigational use. The #HOPE4KIDNEY Trial is designed to support a future expansion of the indication to include the destruction of kidney tissue/tumors. About Alpha Wave Alpha Wave is a global investment company with three main verticals: private equity, private credit, and public markets. It is led by Rick Gerson, Navroz Udwadia, and Ryan Khoury. Its flagship global private equity fund, Alpha Wave Ventures, aims to invest in best-in-class growth-stage companies and endeavors to be helpful long-term partners to the founders and management teams. Alpha Wave has offices in Miami, New York, London, Monaco, Madrid, Abu Dhabi, Tel Aviv, Bangalore, and Sydney. For more information, please visit www.alphawaveglobal.com. For more information please visit: www.histosonics.com/ Contacts For Media Inquiries Media contacts:
Josh King Vice President of Global Market Access Email : Joshua.king@histosonics.com Phone : +1 608.332.8124
Kimberly Ha KKH Advisors 917-291-5744 kimberly.ha@kkhadvisors.com
G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company focused on delivering next-generation therapies that improve the lives of those affected by cancer, and Pharmacosmos A/S, a leader in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anemia, today announced that they have entered into a definitive merger agreement under which Pharmacosmos A/S, through its U.S. subsidiary Pharmacosmos Therapeutics Inc., will acquire all outstanding shares of G1 Therapeutics common stock for U.S. $7.15 per share in cash for a total equity value of approximately $405 million, which represents a 68% premium to G1’s closing share price on August 6, 2024 and a 133% premium to G1’s prior 30-day volume weighted average price. The Boards of Directors of the parties have unanimously approved the transaction, which is expected to close late in the third quarter of 2024.
G1’s COSELA is the first and only product approved by the U.S. Food and Drug Administration to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC).
Together, Pharmacosmos and G1 Therapeutics will execute on the shared vision to grow and accelerate the availability of COSELA for all appropriate patients with ES-SCLC. G1 brings a well-established and successful commercial, sales, and medical platform to Pharmacosmos, which has complementary expertise in commercializing hematology and supportive care products, a robust global commercial presence, and significant resources to maximize the penetration of COSELA into the ES-SCLC market. Together, the combined company will be able to optimize the commercial reach to oncologists and expand the availability of COSELA among patients living with ES-SCLC.
“G1 and Pharmacosmos have a shared commitment to people living with cancer; the transaction announced today will enable a more rapid uptake of COSELA into the ES-SCLC market to maximize availability for patients who need this important drug,” said Jack Bailey, Chief Executive Officer of G1 Therapeutics. “Importantly, this acquisition delivers significant value to G1’s stakeholders by providing better and broader access to this important product for the cancer patients we seek to treat and a significant premium to our shareholders. I am proud of all that the G1 team has accomplished over the years, thankful for their great effort, and excited about what’s possible by the combined Pharmacosmos/G1 team as we meet the needs of more cancer patients.”
“The acquisition of G1 Therapeutics Inc., its intellectual property, and the addition of COSELA® (trilaciclib) to our portfolio of innovative products is transformative for Pharmacosmos. By combining our existing colleagues with the great team at G1 Therapeutics, we will meaningfully expand our organization serving oncologists in the US. This will enable broader and better access for patients in need of COSELA as well as for our existing FDA approved drug, Monoferric® (ferric derisomaltose),” said Tobias S. Christensen, President and Chief Executive Officer of Pharmacosmos A/S. “COSELA is a first-in-class product that brings important benefits to patients and fits very nicely together with our lead product Monoferric® (ferric derisomaltose). While Monoferric is available around the World, COSELA is so far only approved in the US and in China. It will be a focus for us to bring this important product to more patients both in US and worldwide to help minimize the number of lung cancer patients suffering from myelosuppression after chemotherapy.”
Transaction Terms
Under the terms of the merger agreement, Pharmacosmos has agreed to commence a cash tender offer to acquire all issued and outstanding shares of G1 common stock for US $7.15 per share in cash. The transaction will be fully financed by Pharmacosmos’ existing cash on hand and existing corporate credit facilities.
The closing of the tender offer will be subject to customary conditions, including the tender of shares which represent at least a majority of the total number of G1’s outstanding shares of common stock and the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Upon successful completion of the tender offer, Pharmacosmos would acquire all shares not acquired in the tender offer through a second-step merger for the same consideration that the tendering stockholders will receive in the tender offer.
It is anticipated the transaction will close late in the third quarter of 2024. Upon completion of the transaction, G1’s common stock will no longer be publicly listed.
As previously announced, G1 will be releasing its second quarter 2024 financial results and filing its Form 10-Q Quarterly Report tomorrow. However, due to the pending transaction, we will no longer be hosting a conference call at 8:30 am ET, August 8 to review such results.
Advisors
For Pharmacosmos, MTS Health Partners, L.P. is serving as exclusive financial advisor, and Arnold & Porter Kaye Scholer LLP is serving as legal counsel. For G1, Centerview Partners LLC is serving as exclusive financial advisor, and Ropes & Gray LLP and Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. are serving as legal counsel.
About COSELA® (trilaciclib) for Injection
COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.
Indication
COSELA® (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.
Important Safety Information
COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.
Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.
The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.
To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.
About Monoferric (ferric derisomaltose)
Indication
Monoferric (ferric derisomaltose) is indicated for the treatment of iron deficiency anemia (IDA) in adult patients:
who have intolerance to oral iron or have had unsatisfactory response to oral iron
who have non-hemodialysis dependent chronic kidney disease (NDD-CKD)
Important Safety Information
Monoferric is contraindicated in patients with a history of serious hypersensitivity to Monoferric or any of its components. Reactions have included shock, clinically significant hypotension, loss of consciousness, and/or collapse.
Warnings and precautions include serious hypersensitivity reactions, including anaphylactic-type reactions, some of which have been life-threatening and fatal, have been reported in patients receiving Monoferric. Patients may present with shock, clinically significant hypotension, loss of consciousness, and/or collapse. Monitor patients for signs and symptoms of hypersensitivity during and after Monoferric administration for at least 30 minutes and until clinically stable following completion of the infusion. Only administer Monoferric when personnel and therapies are immediately available for the treatment of serious hypersensitivity reactions. Monoferric is contraindicated in patients with prior serious hypersensitivity reactions to Monoferric or any of its components. In clinical trials in patients with IDA and CKD, serious or severe hypersensitivity were reported in 0.3% (6/2008) of the Monoferric treated subjects. These included 3 events of hypersensitivity in 3 patients; 2 events of infusion-related reactions in 2 patients and 1 event of asthma in one patient.
Excessive therapy with parenteral iron can lead to excess iron storage and possibly iatrogenic hemosiderosis or hemochromatosis. Monitor the hematologic response (hemoglobin and hematocrit) and iron parameters (serum ferritin and transferrin saturation) during parenteral iron therapy. Do not administer Monoferric to patients with iron overload.
Adverse reactions were reported in 8.6% (172/2008) of patients treated with Monoferric. Adverse reactions related to treatment and reported by ≥1% of the treated patients were nausea (1.2%) and rash (1%). Adjudicated serious or severe hypersensitivity reactions were reported in 6/2008 (0.3%) patients in the Monoferric group. Hypophosphatemia (serum phosphate <2.0 mg/dL) was reported in 3.5% of Monoferric-treated patients in Trials 1 & 2.
To report adverse events, please contact Pharmacosmos at 1-888-828-0655. You may also contact the FDA at www.fda.gov/medwatch or 1-800-FDA-1088.
Pharmacosmos A/S, headquartered in Holbaek, Denmark, and founded in 1965, is a highly specialised company focused on carbohydrate chemistry and a global leader in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anaemia. With companies in the UK, Ireland, Nordics, Germany, the USA, and China, as well as through partners, Pharmacosmos markets its products around the world. With a strong and ongoing commitment to R&D, Pharmacosmos is able to leverage a unique carbohydrate production platform along with deep expertise in the synthesis of iron-carbohydrate complexes. The Pharmacosmos Group has more than 500 employees.
About G1 Therapeutics
G1 Therapeutics, Inc. is a commercial-stage oncology biopharmaceutical company whose mission is to develop and deliver next-generation therapies that improve the lives of those affected by cancer, including the Company’s first commercial product, COSELA® (trilaciclib). G1’s goal is to provide innovative therapeutic advances for people living with cancer. G1 is based in Research Triangle Park, N.C. For additional information, please visit www.g1therapeutics.com and follow us on X (formerly known as Twitter) @G1Therapeutics and LinkedIn.
G1 Therapeutics® and the G1 Therapeutics logo and COSELA® and the COSELA logo are trademarks of G1 Therapeutics, Inc.
Forward-Looking Statements
This document contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the proposed acquisition of G1 by Pharmacosmos, the expected timetable for completing the transaction, and G1’s future financial or operating performance. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “seek,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this document are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation: (i) risks associated with the timing of the closing of the proposed transaction, including the risks that a condition to closing would not be satisfied within the expected timeframe or at all or that the closing of the proposed transaction will not occur; (ii) uncertainties as to how many of G1’s stockholders will tender their shares in the offer; (iii) the possibility that a governmental entity may prohibit, delay or refuse to grant approval for the consummation of the transaction; (iv) the possibility that competing offers will be made; (v) the outcome of any legal proceedings that may be instituted against the parties and others related to the merger agreement; (vi) unanticipated difficulties or expenditures relating to the proposed transaction, the response of business partners and competitors to the announcement of the proposed transaction, and/or potential difficulties in employee retention as a result of the announcement and pendency of the proposed transaction; (vii) G1’s ability to successfully demonstrate the efficacy and safety of its drug or drug candidates, and the preclinical or clinical results for its product candidates, which may not support further development of such product candidates; (viii) comments, feedback and actions of regulatory agencies; (ix) G1’s dependence on the commercial success of COSELA (trilaciclib); (x) the inherent uncertainties associated with developing new products or technologies and operating as commercial stage company; (xi) chemotherapy shortages; and (xii) other risks identified in G1’s SEC filings, including G1’s Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent filings with the SEC. G1 cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. G1 disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Additional Information and Where to Find It
The tender offer referred to in this document has not yet commenced. This document is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell shares, nor is it a substitute for the tender offer materials that Pharmacosmos and its acquisition subsidiary will file with the SEC upon commencement of the tender offer. At the time the tender offer is commenced, Pharmacosmos and its acquisition subsidiary will cause to be filed a tender offer statement on Schedule TO with the SEC, and G1 will file a solicitation/recommendation statement on Schedule 14D-9 with respect to the tender offer. THE TENDER OFFER STATEMENT (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND OTHER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT WILL CONTAIN IMPORTANT INFORMATION THAT SHOULD BE READ CAREFULLY AND CONSIDERED BY G1’S STOCKHOLDERS BEFORE ANY DECISION IS MADE WITH RESPECT TO THE TENDER OFFER. Both the tender offer statement and the solicitation/recommendation statement will be mailed to G1’s stockholders free of charge. A free copy of the tender offer statement and the solicitation/recommendation statement will also be made available to all stockholders of G1 by accessing https://investor.g1therapeutics.com/ or by contacting Investor Relations at ir@g1therapeutics.com. In addition, the tender offer statement and the solicitation/recommendation statement (and all other documents filed with the SEC) will be available at no charge on the SEC’s website: www.sec.gov, upon filing with the SEC.
G1’S STOCKHOLDERS ARE ADVISED TO READ THE SCHEDULE TO AND THE SCHEDULE 14D-9, AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME, AND ANY OTHER RELEVANT DOCUMENTS FILED WITH THE SEC WHEN THEY BECOME AVAILABLE BEFORE THEY MAKE ANY DECISION WITH RESPECT TO THE TENDER OFFER, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION AND THE PARTIES THERETO.
Pharmacosmos A/S Contact:
Christian Lundquist Madsen VP Global Marketing & Communication +45 5948 5959 clm@pharmacosmos.com
G1 Therapeutics Contacts:
John W. Umstead V Chief Financial Officer 919-747-8419 jumstead@g1therapeutics.com
Will Roberts Communications Officer Vice President, Investor Relations and Corporate Communications (919) 907-1944 wroberts@g1therapeutics.com
One of our Fierce 15 honorees dove deep into the ocean for inspiration from the sea squirt to develop new human therapeutics. Another thought the adorable 13-lined ground squirrel, with its polka dots and hibernation habits, might provide a clue to new treatments.
And there’s the biotech that looked at the Human Genome Project and thought, what else can it do?
There’s a company looking at psychedelics in a whole new way, rising on the recent interest in repurposing the illicit substances for something better, like improving mental health.
And then, over in New Zealand, one tiny company is levying a fight against viral infections. It has been told its mission is outrageous, but we don’t think so.
Welcome to the 2024 Fierce 15. The Fierce Biotech editorial team is proud to present this class of the best in biotech. These 15 companies were carefully selected from hundreds of nominees.
This is the hardest project we do all year—to pick just 15 companies breaking barriers and defining the future of the industry. But it’s also the most fulfilling when we’re done and the most exciting to publish. So read on to find out who we picked this year.
Cour Pharmaceuticals
Retraining the culprits at the center of autoimmune diseases
What makes Cour fierce: Cour Pharmaceuticals hopes to bring more than four decades’ worth of research into the immune system to fruition with a novel type of reverse vaccine that aims to reprogram the body’s aggressive—if a touch overzealous—white blood cells and ultimately convince them to not attack otherwise healthy organs.
The startup’s goal is to build a targeted, platform-based approach that can address debilitating autoimmune diseases without blunting a patient’s ability to fight off an invading infection—and one that could potentially be tailored for a variety of different conditions.
With technology originally developed at the Northwestern University Feinberg School of Medicine in Chicago, Cour employs biodegradable nanoparticles that encapsulate certain antigens—specifically, whichever peptides or proteins are needed to match the ones responsible for triggering the chain reaction that drives T cells to destroy the body’s own tissues.
These nanoparticles hitch a ride on white blood cells known as monocytes as they travel to the spleen and liver. When those cells are recycled by the body, the antigens are released in a way that makes them look like normal dead cellular debris, which the immune system then learns to recognize.
Where a traditional vaccine would instruct these types of cells to attack a particular intruder, Cour’s strategy is to assure the immune system that they’re allowed to pass by unharmed.
“It was a 40-year endeavor trying to figure out how you get the immune system to be retrained so that it won’t attack the antigen of interest,” CEO John Puisis said in an interview with Fierce. “We are just very fortunate to hit the crossroads of a serendipitous scientific medical discovery.”
In the earliest stages of the company, the tactic was studied as a way to tamp down the dangerous inflammatory responses seen in people with severe peanut allergies. Today, Cour is focused solely on autoimmunity, with a pipeline that includes candidates targeting Type 1 diabetes, celiac disease and the muscle-weakening condition myasthenia gravis. And, so far, early results have been promising, according to the CEO.
“We can actually abrogate the disease,” Puisis said. “That’s what our data shows, which is really profound.”
In Type 1 diabetes, where T cells assault the insulin-producing islet cells of the pancreas, Cour plans to halt the progression of the disease.
The biotech’s CNP-103 program aims to present the body with 120 antigen epitopes by encapsulating four different recombinant proteins at once—and it’s slated to make a first-in-human debut next year through Cour’s phase 1b/2a trial.
The plan is to start the study with participants in the advanced stages of the disease—namely those with established losses of pancreatic beta cells who may be considering a tissue transplant, despite still having the autoimmunity—before developing it into a treatment for early diagnoses.
“Type 1 diabetes is still not terribly well managed today, despite improvements and continuous glucose monitoring,” said Chief Operating Officer Dannielle Appelhans. “But for addressing the underlying cause, there’s really nothing available.”
Meanwhile, Cour’s CNP-106 treatment for myasthenia gravis has cleared the proof-of-concept stage and is enrolling adults in a similar early to midphase study.
For celiac disease, the company has found a partner in Takeda, with a global licensing agreement inked in 2019 that includes a commitment of up to $420 million in potential milestone and royalty payments. That therapy—known as CNP-101 or TAK-101, depending on who you ask—smuggles in the gliadin proteins that serve as one of the two major components of gluten compounds.
In a previous first-in-human clinical trial of about 30 patients, the company demonstrated that within the weeks following two infusions, measures of inflammatory cytokine biomarkers as well as T-cell activation and infiltration into the small intestine were greatly diminished, even when study participants were subsequently given large doses of gluten.
“Doctors who diagnose patients with celiac disease give them a gluten challenge, and then they go look at the T cells,” Puisis said.
“What a few doctors told us was that, with our treatment, that person would no longer have been diagnosed. So take that for what it’s worth, but that’s a pretty profound change, and it was significant with a very small population,” he said. “I’m being cautiously exuberant.”
At the same time, Cour expects its products will be cheaper compared to gene therapies, despite falling under the same regulatory category as other cell-programming tech.
“What’s really interesting about what we’re doing is that we have this platform, but 98% of it is a polymer, and then the proteins are only 1% to 2%,” said Appelhans, who previously served as COO and CEO of cell therapy developer Rubius Therapeutics and as chief technical officer of Novartis’ gene therapy division. Appelhans will take over the CEO role from Puisis effective Sept. 1.
“So what really excites me is the potential to reach many patients across the world, because, while being an advanced modality, it doesn’t have the same cost structure associated with those.”
COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that as part of a leadership succession plan, John J. Puisis will transition his responsibilities as President and Chief Executive Officer of COUR to the Company’s Chief Operating Officer, Dannielle Appelhans, effective September 1, 2024. Mr. Puisis, who founded and led COUR through multiple INDs, clinical studies and strategic partnerships, will continue to serve in his role on the Company’s Board of Directors and will be an advisor to COUR through the end of 2024. Ms. Appelhans has also been appointed to the COUR Board of Directors.
“Leading COUR since its inception over a decade ago has been a highlight of my career,” stated Mr. Puisis. “With the support of industry-leading partners and top-tier investors, the COUR team has transformed a serendipitous discovery in the labs at Northwestern University into a clinically validated nanoparticle platform poised to deliver first-in-class, disease-modifying treatments for autoimmune and inflammatory diseases. Since securing our $105 million Series A earlier this year, we’ve solidified the Company’s foundation by filling critical management roles, expanding our board of directors, and fortifying key partnerships. The Company is now stronger than ever before. Given Dannielle’s significant contributions since joining COUR in 2023, and her extensive experience in the biopharmaceutical industry, I am confident in her vision for COUR’s future and her ability to lead the Company as CEO through the next phases of growth and expansion. I look forward to collaborating with Dannielle to ensure a seamless transition of leadership responsibilities and am excited to serve alongside her on COUR’s Board of Directors.”
Ms. Appelhans joined COUR as COO in 2023 and has since led R&D, technical development, clinical operations, quality, technical operations and has played a pivotal role in shaping recent corporate strategy. Before joining COUR, she served as CEO of Rubius Therapeutics, where she previously held the position of COO. Since 2022, she has served on the Board of Directors and as a member of the Nominating and Governance Committee for Generation Bio Co. Prior to Rubius, Ms. Appelhans held progressive roles at Novartis, culminating in her tenure as Senior Vice President, Head of Global Supply Chain across all divisions, and later as Senior Vice President of Technical Operations and Chief Technical Officer at Novartis Gene Therapies, where she oversaw late-stage development, clinical and commercial manufacturing, supply chain, and all supporting operational functions for the gene therapy unit. Earlier in her career, she served as a Senior Engagement Manager at McKinsey & Company and held roles of increasing responsibility at Eli Lilly and Company.
Ms. Appelhans earned a Master of Science in mechanical engineering from Massachusetts Institute of Technology’s School of Engineering, an MBA from Massachusetts Institute of Technology’s Sloan School of Management, and a Bachelor of Science in Engineering in mechanical engineering from the University of Michigan.
Ms. Appelhans remarked, “I joined COUR in 2023 because I saw tremendous potential in the Company’s platform technology. After over a year of working closely with the team, it’s evident to me that our nanoparticle approach to antigen-specific immune tolerance is at the forefront of the industry. As CEO, I am eager to build upon the momentum of our recent achievements, expanding COUR’s portfolio and advancing our clinical programs to pivotal stages, with the ultimate aim of delivering new life-changing treatments to patients.”
“On behalf of the COUR Board of Directors, I extend heartfelt gratitude to John for his more than 10 years of dedicated service to COUR Pharmaceuticals. Under his leadership, COUR has established extensive capabilities and assembled a strong leadership team laying the groundwork for future success. John’s contributions have been instrumental in shaping COUR into the organization it is today,” said Robert F. Carey, Lead Independent Director of the COUR Board of Directors. “Looking ahead, we are excited to announce Ms. Appelhans’ transition to the role of CEO. With our recent fundraising success and the anticipated clinical progress of our wholly owned programs for Myasthenia Gravis and Type 1 Diabetes, we believe COUR is well-positioned for continued success.”
About COUR Pharmaceuticals:
COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune and inflammatory diseases.
COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having partnered products in Celiac Disease (with Takeda Pharmaceuticals), and Primary Biliary Cholangitis (with Ironwood Pharmaceuticals).
X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced positive new clinical data from its ongoing Phase 2 clinical trial evaluating the safety and efficacy of mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia (CN). An interim analysis of data from the ongoing six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (ANC) both as a monotherapy and in combination with stable doses of injectable granulocyte colony-stimulating factor (G-CSF), the only therapy approved in the U.S. for severe chronic neutropenia.
Today the company also announced that it is currently screening patients for enrollment into its global, pivotal Phase 3 clinical trial, the 4WARD study, evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants.
“For the first time, we have demonstrated the ability of mavorixafor monotherapy to durably and meaningfully increase ANC in people living with chronic neutropenia,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “In addition, we were pleased to see that once-daily mavorixafor used in combination with G-CSF also led to meaningful and sustained ANC increases and was generally well tolerated, further supporting the design of our newly initiated Phase 3 4WARD clinical trial, which will study the use of mavorixafor alone and with G-CSF in people with CN dealing with recurrent and/or serious infections. Today’s exciting interim results build on the momentum we established in April with mavorixafor’s first approval in the U.S., and we look forward to quickly advancing the 4WARD trial as we pursue a second indication to deliver for more patients in need.”
Interim Analysis of Data from Phase 2 Clinical Study of Mavorixafor in CN The Phase 2 study of mavorixafor is a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic CN. The interim analysis included results from the two treatment groups in the study (mavorixafor monotherapy and mavorixafor with stable-dose G-CSF) that most closely mirror the participant population of the newly initiated Phase 3 4WARD trial. Fifteen participants were enrolled across these two groups and, as of the May 14, 2024 interim analysis data cut-off date, seven had completed the study, and five remain ongoing. Data from a third treatment group of eight participants receiving mavorixafor and dose-adjusted G-CSF are expected to be presented later this year.
The mavorixafor monotherapy group included 10 participants and the mavorixafor with stable-dose G-CSF group included five participants. As of the data cut-off date, findings from the interim analysis show:
100% (6/6) of evaluable participants who had completed the six-month study achieved target ANC increase (ANC >500 cells/µL) at Months 3 and 6 on once-daily mavorixafor therapy with or without stable-dose G-CSF.
Participants on mavorixafor monotherapy achieved mean ANC levels above the lower limit of normal for CN (≥1,500 cells/µL) at Month 3 (n=8) and Month 6 (n=3).
Mavorixafor monotherapy also durably increased ANC in participants with severe CN (ANC<500 cells/µL at baseline), achieving mean ANC of ~800-1,000 cells/µL (ANC range targeted by experts) at Months 1, 3, and 6 (n=5, 3, and 2, respectively).
Participants on mavorixafor in combination with stable-dose G-CSF experienced increases in mean ANC of >1,000 cells/µL at Months 1, 3, and 6 (n=4, 4, and 3, respectively) versus baseline.
Across the 23 participants enrolled in the study, mavorixafor was generally well tolerated as a monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported, as of the interim analysis data cut-off date. Of the 23 participants, three discontinued due to non-serious adverse events. The overall safety profile remains consistent with previous clinical studies.
Jean Donadieu, M.D., Ph.D., pediatrician in the hemato-oncology department of Trousseau Hospital in Paris, coordinator of the French registry for chronic neutropenia, and coordinator of the French chronic neutropenia reference center, commented on the results: “I am pleased to see that these interim data are consistent with the previous results of the Phase 1b study, but now with durability of effect and a good tolerability profile out to six months of treatment. This patient group has only one currently approved treatment option – one that is injectable and that has dose-related, dose-limiting, and challenging side effects and risks. The results from this interim analysis offer a sound and compelling rationale for mavorixafor’s evaluation in a Phase 3 trial, which is very good news for my patients with chronic neutropenia who, I believe, would benefit from an oral therapy to help reduce recurring or severe infections.”
Investor Webinar Details: X4 will host an investor webinar to present and discuss the new data today at 8:00 am ET. To register for the event, click here. A live Q&A will follow the formal presentation. Following the conclusion of the live webcast, a replay of the event and the presented slides will be available within the investors’ section of the X4 Pharmaceuticals website at www.x4pharma.com.
About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a rare blood condition lasting more than three months, persistently or intermittently, and characterized by increased risk of infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC).
About the Phase 1b/Phase 2 Chronic Neutropenia Trial The Phase 1b/Phase 2 clinical trial (NCT04154488) is a proof-of-concept, open-label, multicenter study designed to assess the safety and tolerability of oral mavorixafor, with or without injectable G-CSF, in participants with chronic neutropenic disorders, including idiopathic, cyclic, and congenital neutropenia. In the Phase 1b portion of the study, participants received one dose of oral mavorixafor and were assessed for magnitude of absolute neutrophil count (ANC) response and tolerability. In this initial portion of the study, 100% of participants (n=25) responded to treatment and mavorixafor was generally well tolerated alone or dosed concurrently with G-CSF. The ongoing Phase 2 portion of the trial (n=23 fully enrolled) is assessing the safety, tolerability, and the impact on participants’ neutropenia of oral, once-daily mavorixafor with and without concurrent injectable G-CSF therapy over a six-month period.
About the 4WARD Global, Pivotal, Phase 3 Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants with confirmed trough ANC levels less than 1,500 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response.
About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI™ (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.
Forward-Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the potential therapeutic benefit of mavorixafor; the initiation, timing, progress, and results of our current and future studies and clinical trials, including the Phase 2 clinical trial in chronic neutropenia and the Phase 3 4WARD clinical trial and related preparatory work and the period during which the results of the trials will become available; and the mission and goals for our business. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: we may encounter adverse events for mavorixafor at any stage that negatively impact development and/or commercialization; the expected availability, content, and timing of clinical data from our ongoing clinical trials of mavorixafor may be delayed or unavailable, including clinical results from our ongoing Phase 2 clinical trial and the announced Phase 3 4WARD trial; the trials and studies may not have satisfactory outcomes; the outcomes of preclinical studies or earlier clinical trials will not be predictive of later clinical trial results; the design and rate of enrollment for clinical trials, including the current design of our Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders may not enable successful completion of the trial(s); we may be unable to obtain and maintain regulatory approvals; uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; initial or interim results from a clinical trial may not be predictive of the final results of the trial or the results of future trials; the potential adverse safety effects arising from the testing or use of our product and product candidates may negatively impact development and/or commercialization; there will be changes in expected or existing competition; there will be changes in the regulatory environment; our business may be adversely affected and our costs may increase if any of our key collaborators fails to perform its obligations or terminates our collaboration; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause us to use cash more quickly than we expect or to change or curtail some of our plans or both; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2024, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.
Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced final results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD). Final results from this study confirmed the excellent safety profile of TPN-101 and showed clinical signs of disease-modifying effects in patients with these disorders, which align with previously reported safety and therapeutic activity in patients with Progressive Supranuclear Palsy (PSP). These findings are consistent with TPN-101’s reduction of neuroinflammation and neurodegeneration through blocking the activity of LINE-1, a human-specific retrotransposon that is no longer adequately suppressed in many neurodegenerative disorders and aging.
ALS is a progressive and uniformly fatal neurodegenerative disease with a mean survival of two-to-three years. Respiratory failure is the most common cause of death for people with ALS. Vital Capacity (VC) is an objective measure of respiratory function that correlates with mortality in these patients. After 24 weeks, C9-ALS participants treated with TPN-101 experienced approximately 50% less decline in VC compared to those treated with placebo (LS mean change -8.4% vs -16.5%). When the participants in the placebo group were switched to TPN-101 during the open-label period, the decline in VC over the following 24 weeks (-7.2%) was less than half that while on placebo and comparable to that in the original TPN-101 group during the double-blind period. Overall, the 48-week changes in both groups were lower than expected based on natural history in similar study populations.
The decline on the Revised ALS Functional Rating Scale (ALSFRS-R) was comparable between the TPN-101 and placebo groups during the 24-week double-blind period (LS mean -7.2 points vs -6.7). However, during the 24-week open-label extension period, the decline on the ALSFRS-R in the original TPN-101 group was less than half that during the initial 24-week period and less than half that of the placebo group during both study periods. The decline in ALSFRS-R in the original TPN-101 group over the entire 48 weeks was approximately 40% less than expected based on natural history data, indicating a global clinical benefit with longer treatment.
“The effects of TPN-101 across multiple key endpoints in this study are encouraging and represent an important step forward in finding a potential treatment for this serious illness,” said Merit Cudkowicz, M.D., Chair of the Massachusetts General Hospital Department of Neurology, Julieanne Dorn Professor of Neurology at Harvard Medical School, and principal investigator in the Phase 2 study of TPN-101 for C9orf72-related ALS/FTD. “I look forward to advancing the development of TPN-101 and what that could mean for people living with C9-ALS.”
In participants with C9-ALS, those treated with TPN-101 had lower levels of neurofilament light chain (NfL) compared with placebo at the end of the double-blind period. NfL is the primary biomarker of neurodegeneration, and the NfL results at both Weeks 24 and 48 are consistent with findings from the company’s Phase 2 study of TPN-101 for the treatment of PSP. TPN-101 also had lowering effects on additional biomarkers of neurodegeneration and neuroinflammation, including neurofilament heavy chain (NfH), interleukin 6 (IL-6), neopterin, and osteopontin.
A meta-analysis of the combined C9-ALS and PSP populations from the two Phase 2 studies of TPN-101 showed a statistically significant NfL-lowering effect of TPN-101 versus placebo at Week 24 (p = 0.034). The consistency of biomarker improvements in these two studies evaluating patients with different clinical conditions supports the treatment hypothesis that these diseases share a common LINE-1-related pathophysiology.
“Today there are very limited treatment options for ALS patients and based on these results that show patients treated with TPN-101 experience impactful benefits across multiple functional and biomarker measures, we plan to rapidly advance TPN-101 into a Phase 3 registration study for the treatment of C9-ALS,” said Dennis Podlesak, Chairman and Chief Executive Officer of Transposon. “In addition to ALS, we are committed to advancing TPN-101 for the treatment of PSP, Alzheimer’s disease and other neurodegenerative and autoimmune disorders with the goal of providing new and innovative therapies that can significantly improve the lives of those battling these devastating diseases.”
Transposon intends to present detailed data from this study at upcoming scientific meetings.
About the Phase 2 Study in C9orf72-related ALS/FTD
The Phase 2 study in C9orf72-related ALS/FTD is multi-center, randomized, double-blind, placebo-controlled parallel-group, two-arm study with an open-label treatment phase in patients with C9orf72-related ALS and/or FTD. Participants (n=42) were randomized to receive daily doses of 400 mg of TPN-101 or placebo. The study includes a six-week screening period, a 24-week double-blind treatment period, a 24-week open label treatment period, and a follow-up visit four weeks post-treatment. All phases of the study, including the 24-week open label treatment period, have been completed. Further information on the study can be accessed at ClinicalTrials.gov.
About TPN-101
TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering innate immune responses that contribute to neurodegenerative, autoimmune and aging-related disease pathology.
About ALS and FTD
ALS is a neurodegenerative disease characterized by progressive muscle weakness, loss of ability to speak, eat, move or breathe. FTD is a progressive frontal / temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating or working. With onset commonly in middle age or earlier, patients with ALS have a mean survival of two-to-three years. Patients with FTD have a mean survival of nine years.
About Transposon
Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications.
Contact: Rick Orr Transposon Therapeutics, Inc. (858) 535-4821 rorr@transposonrx.com
Edwards Lifesciences (NYSE: EW) today announced investments that reflect the company’s deep commitment to advancing patient care through structural heart innovation, addressing large unmet patient needs and supporting sustainable long-term growth.
Edwards has entered into an agreement to acquire JenaValve Technology, a pioneer in the transcatheter treatment of aortic regurgitation (AR), a deadly disease that impacts a significant and growing population and is largely untreated today. JenaValve presented positive results of its U.S. pivotal trial for the treatment of symptomatic, severe AR in high-risk patients late last year. As the pioneer in valve innovation for more than 60 years, Edwards believes it is uniquely positioned to lead this next frontier of aortic valve disease treatment. Edwards anticipates FDA approval of the JenaValve Trilogy Heart Valve System in late 2025, which will represent the first approved therapy for patients suffering from AR.
Building on an investment made in 2016, Edwards has exercised its option to acquire Endotronix, a leader in heart failure (HF) management solutions. Many structural heart patients Edwards serves today also suffer from HF with limited options. This acquisition will expand Edwards’ structural heart portfolio into a new therapeutic area to address the large unmet needs of patients suffering from HF. Last month, Endotronix received FDA approval for Cordella, an implantable pulmonary artery pressure sensor allowing early, targeted therapeutic intervention. A CMS national coverage determination is expected in early 2025.
“These acquisitions expand our opportunities to address the unmet needs of aortic regurgitation and heart failure patients around the world,” said Bernard Zovighian, Edwards’ CEO. “We are pleased to enter these structural heart therapeutic areas with innovation, world-class science and clinical evidence to provide access to life-saving technologies for patients around the world.”
Edwards anticipates these investments will strengthen its leadership in structural heart innovation and represent long-term growth opportunities. Edwards expects minimal revenue contribution from these acquisitions in 2025. The aggregate upfront purchase price for these strategic investments is approximately $1.2 billion. The acquisitions are subject to the satisfaction of certain closing conditions, including the receipt of required antitrust and foreign investment approvals.
About Edwards Lifesciences
Edwards Lifesciences is the global leader of patient-focused innovations for structural heart disease and critical care monitoring. We are driven by a passion for patients, dedicated to improving and enhancing lives through partnerships with clinicians and stakeholders across the global healthcare landscape. For more information, visit www.edwards.com and follow us on Facebook, Instagram, LinkedIn, X and YouTube.
This news release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. We intend the forward-looking statements contained in this press release to be covered by the safe harbor provisions of such Acts. These forward-looking statements can sometimes be identified by the use of forward-looking words, such as “may,” “might,” “believe,” “will,” “expect,” “project,” “estimate,” “should,” “anticipate,” “plan,” “goal,” “continue,” “seek,” “intend,” “optimistic,” “aspire,” “confident” and other forms of these words and include, but are not limited to, statements made by Mr. Zovighian and statements regarding our expected continued performance of Edwards; performance of the Edwards, JenaValve or Endotronix technologies; product and therapy benefits; patient access and outcomes; size of treatable population; leading position; growth opportunities; unmet needs in structural heart, aortic regurgitation, and heart failure therapeutic areas; probability of approval by the FDA and in the anticipating timeline; probability of a positive NCD by the CMS and in the anticipated timeline; synergies between the technologies, business, and operations of each of JenaValve and Endotronix and Edwards’ technologies, products, portfolio, expertise, and operations; ability to leverage the technology or innovation from these acquisitions or cause or ensure accelerated access to life-saving technologies for patients or development of novel technologies as a result of these acquisitions; commitment to expand opportunities in structural heart innovation, address large unmet patient needs, and support sustainable long-term growth; objective to expand Edwards’ portfolio into new structural heart therapeutic areas; opportunities and revenue return on these acquisitions and their contribution to Edwards’ growth and performance, as well as the expectations on timing of such returns and contributions; therapy approval pipeline for patients suffering from AR; probability of the closing of the two acquisitions; other objectives and expectations; and other statements that are not historical facts. Forward-looking statements are based on estimates and assumptions made by management of the company and are believed to be reasonable, though they are inherently uncertain and difficult to predict. Our forward-looking statements speak only as of the date on which they are made, and we do not undertake any obligation to update any forward-looking statement to reflect events or circumstances after the date of the statement. Investors are cautioned not to unduly rely on such forward-looking statements.
Forward-looking statements involve risks and uncertainties that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements. Factors that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements include, but are not limited to: (i) Edwards may be unable to close the acquisitions of each of JenaValve and Endotronix, which may materially and adversely affect Edwards’ business and the price of Edwards’ common stock; (ii) the occurrence of any event, change or other circumstance that could cause Edwards to abandon the acquisitions of either or both of JenaValve and Endotronix; (iii) risks related to disruption of management’s attention from Edwards’ ongoing business operations; (iv) the effect of the announcement or the pendency of the acquisitions on Edwards’ relationships with its customers, operating results and business generally; (v) potential significant transaction costs associated with either or both acquisitions; (vi) the outcome of any legal proceedings or regulatory actions to the extent initiated against Edwards or others related to either or both acquisitions; (vii) the ability of Edwards to execute on its strategy and achieve its goals and other expectations after the closing of either or both acquisitions; (viii) legal, regulatory, tax and economic developments affecting Edwards’ business; (ix) the unpredictability and severity of catastrophic events, including, but not limited to, acts of terrorism, outbreak of war or hostilities or current or future pandemics or epidemics, as well as Edwards’ response to any of the aforementioned factors; and (x) other risks detailed in Edwards’ filings with the SEC, which may be found at edwards.com.
Edwards, Edwards Lifesciences, and the stylized E logo are trademarks of Edwards Lifesciences Corporation or its affiliates. All other trademarks are the property of their respective owners.
Contacts
Media Contact: Amy Hytowitz, 949-250-4009 Investor Contact: Mark Wilterding, 949-250-6826
Toronto (Head Office)
141 Adelaide Street
Suite 770
Toronto, ON
Canada, M5H 3L5
Montréal
Espace CDPQ
3, Place Ville Marie
Bureau 12350, Niveau L
Montreal, QC
Canada, H3B 0E7
